Trial Outcomes & Findings for Safety, Pharmacokinetics and Efficacy of Paxalisib (GDC-0084) in Newly-diagnosed Glioblastoma (NCT NCT03522298)
NCT ID: NCT03522298
Last Updated: 2025-03-24
Results Overview
A DLT was defined as a Grade 3 or 4 toxicity occurring within the DLT assessment window and assessed to be probably or possibly related to paxalisib. DLTs were graded using the Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. CTCAE Grade 3 is a severe adverse event (AE) and Grade 4 is a life-threatening or disabling AE. DLTs were collected to determine the maximum tolerated dose (MTD), which was defined as the dose level below the dose at which less than 33% of participants experienced a DLT.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Cycle 1, Days 1-28
Results posted on
2025-03-24
Participant Flow
Open-label, multicenter dose-escalation study with expansion. This study comprised 2 stages, Stage 1 (dose escalation) and Stage 2 (dose expansion and fed/fasted investigation)
Each stage started with a screening period (Days -28 to -1), followed by treatment and follow-up periods
Participant milestones
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach
|
Dose-escalating Cohort 2: Paxalisib 75 mg
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
6
|
10
|
11
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
6
|
10
|
11
|
Reasons for withdrawal
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach
|
Dose-escalating Cohort 2: Paxalisib 75 mg
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
1
|
|
Overall Study
Death
|
2
|
4
|
8
|
8
|
|
Overall Study
Patient decision
|
0
|
2
|
2
|
2
|
Baseline Characteristics
Safety, Pharmacokinetics and Efficacy of Paxalisib (GDC-0084) in Newly-diagnosed Glioblastoma
Baseline characteristics by cohort
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
68 years
STANDARD_DEVIATION 15.72 • n=99 Participants
|
56 years
STANDARD_DEVIATION 9.30 • n=107 Participants
|
56 years
STANDARD_DEVIATION 12.51 • n=206 Participants
|
59.5 years
STANDARD_DEVIATION 9.46 • n=7 Participants
|
58.5 years
STANDARD_DEVIATION 11.16 • n=31 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
21 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
28 Participants
n=31 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
25 Participants
n=31 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=99 Participants
|
6 participants
n=107 Participants
|
10 participants
n=206 Participants
|
11 participants
n=7 Participants
|
30 participants
n=31 Participants
|
|
Time since diagnosis
|
3.68 months
STANDARD_DEVIATION 0.553 • n=99 Participants
|
4.33 months
STANDARD_DEVIATION 1.137 • n=107 Participants
|
3.64 months
STANDARD_DEVIATION 0.358 • n=206 Participants
|
3.56 months
STANDARD_DEVIATION 0.318 • n=7 Participants
|
3.75 months
STANDARD_DEVIATION 0.637 • n=31 Participants
|
|
Type of surgery
Biopsy (Patients later had a complete resection)
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
|
Type of surgery
Complete resection
|
2 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
22 Participants
n=31 Participants
|
|
Type of surgery
Partial resection
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=31 Participants
|
|
Type of surgery
Other (stereotactic biopsy)
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
|
Histological diagnosis
|
3 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
11 Participants
n=7 Participants
|
30 Participants
n=31 Participants
|
|
Karnofsky Performance Status
100
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=31 Participants
|
|
Karnofsky Performance Status
90
|
0 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
10 Participants
n=7 Participants
|
19 Participants
n=31 Participants
|
|
Karnofsky Performance Status
80
|
3 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=31 Participants
|
|
Karnofsky Performance Status
70
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=31 Participants
|
|
Karnofsky Performance Status
60
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Karnofsky Performance Status
50
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
|
Karnofsky Performance Status
40 or less
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
PRIMARY outcome
Timeframe: Cycle 1, Days 1-28Population: All participants in Stage 1 (dose-escalating cohorts 1 and 2) who received at least one dose of paxalisib
A DLT was defined as a Grade 3 or 4 toxicity occurring within the DLT assessment window and assessed to be probably or possibly related to paxalisib. DLTs were graded using the Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. CTCAE Grade 3 is a severe adverse event (AE) and Grade 4 is a life-threatening or disabling AE. DLTs were collected to determine the maximum tolerated dose (MTD), which was defined as the dose level below the dose at which less than 33% of participants experienced a DLT.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs)
|
0 Participants
|
2 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
The toxicity assessments were made according to the Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A TEAE is any AE occurring or worsening on/after the first study drug dose and within 28 days after the last dose date. The number of participants with Grade 1 to 5 TEAEs are reported here. Specific Adverse Event terms are provided in the Adverse Event module
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (TEAEs)
|
3 Participants
|
6 Participants
|
10 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
An SAE is any AE that meets one or more of the following: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; requires intervention to prevent permanent impairment or damage. Specific AE terms are provided in the Adverse Event module
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Incidence of Serious Adverse Events (SAEs)
|
2 Participants
|
5 Participants
|
6 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
Treatment emergent Adverse Events were graded using the Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. CTCAE Grade 3 is a severe adverse event (AE) and Grade 4 is a life-threatening or disabling AE. Participants are counted at the highest grade TEAE that they experienced.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Incidence of Treatment-emergent Grade 3/4 Treatment Emergent Adverse Events
Grade 3
|
3 Participants
|
3 Participants
|
9 Participants
|
10 Participants
|
|
Incidence of Treatment-emergent Grade 3/4 Treatment Emergent Adverse Events
Grade 1 or 2
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Grade 3/4 Treatment Emergent Adverse Events
Grade 4
|
0 Participants
|
3 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
A change in ECG parameter QTc was defined as an increase of QTc to a value ≥ 500 msec or a change from baseline of at least 60 msec.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Number of Participants Who Experienced a Change in Electrocardiogram (ECG) Parameter QTc
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
A change in left ventricular ejection fraction (LVEF) was defined as a reduction in LVEF to a value of ≤ 45% from baseline.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Number of Participants Who Experienced a Change Left Ventricular Ejection Fraction
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Intention to treat: All patients who were enrolled in the study, whether in Stage 1 or Stage 2.
Progression-free survival is defined as the duration of time from start of treatment to time of progression or death, whichever comes first.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Progression-free Survival Interval Using Using mRANO Criteria/Investigator Review.
|
14.5 Months
Interval 3.3 to 25.7
|
4.7 Months
Interval 1.8 to 27.4
|
7.0 Months
Interval 1.0 to 13.2
|
5.5 Months
Interval 2.0 to 11.8
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Intention to treat: All patients who were enrolled in the study, whether in Stage 1 or Stage 2.
Overall survival is defined as the duration of time from the first day of study treatment until the date of death.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Overall Survival Using RANO Criteria.
|
20.1 Months
Interval 14.4 to 25.7
|
11.8 Months
Interval 4.4 to 37.5
|
12.0 Months
Interval 7.6 to 17.6
|
9.8 Months
Interval 2.0 to 15.1
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 hoursPopulation: Safety population: All participants who received at least 1 dose of paxalisib
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method. Samples were obtained: prior to the initial dose on Cycle 1 Day 1 and then at 30 minutes, and at 1, 2, 3, 4, 6 8 and 24 hours post dose.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Pharmacokinetics of Paxalisib as Area Under the Curve From Time 0 to Last Measurable Time Point (AUC0-last) and/or Area Under the Curve From Time 0 to Infinity (AUC0-inf).
|
1180 ng/mL*h
Geometric Coefficient of Variation 36.6
|
1120 ng/mL*h
Geometric Coefficient of Variation 91.4
|
2190 ng/mL*h
Geometric Coefficient of Variation 34.5
|
1640 ng/mL*h
Geometric Coefficient of Variation 67.1
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 hoursPopulation: Safety population: All participants who received at least 1 dose of paxalisib
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method. Samples were obtained: prior to the initial dose on Cycle 1 Day 1 and then at 30 minutes, and at 1, 2, 3, 4, 6 8 and 24 hours post dose.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Maximum Observed Plasma Concentration of Paxalisib (Cmax)
|
174 ng/mL
Geometric Coefficient of Variation 108
|
118 ng/mL
Geometric Coefficient of Variation 67.4
|
176 ng/mL
Geometric Coefficient of Variation 24.2
|
114 ng/mL
Geometric Coefficient of Variation 44.2
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 hoursPopulation: Safety population: All participants who received at least 1 dose of paxalisib
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method. Samples were obtained: prior to the initial dose on Cycle 1 Day 1 and then at 30 minutes, and at 1, 2, 3, 4, 6 8 and 24 hours post dose.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Pharmacokinetics of Paxalisib as Time to Reach Cmax (Tmax).
|
3.00 Hours
Interval 2.0 to 5.42
|
2.50 Hours
Interval 1.95 to 4.0
|
4.00 Hours
Interval 2.8 to 8.0
|
3.93 Hours
Interval 3.0 to 8.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cycle 1, Day 3Population: All participants in Stage 2 (dose-expansion cohorts) who received at least one dose of paxalisib and had measurable disease
FDG-PET imaging was obtained using a stand-alone PET or combined PET/computed tomography (CT) scanner, or hybrid PET/MRI systems and the and mean FDG-PET standard uptake value quantified
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=5 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=5 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Change in FDG-PET Uptake in Response to Paxalisib in Patients With Measurable Disease.
|
0.036 standardized uptake value
Standard Deviation 0.7523
|
-0.442 standardized uptake value
Standard Deviation 0.3517
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Cycle 1, Day 7Population: All participants in Stage 2 (dose-expansion cohorts) who received at least one dose of paxalisib and had measurable disease
FDG-PET imaging was obtained using a stand-alone PET or combined PET/computed tomography (CT) scanner, or hybrid PET/MRI systems and the and mean FDG-PET standard uptake value quantified
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=5 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=5 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Change in FDG-PET Uptake in Response to Paxalisib in Patients With Measurable Disease
|
-0.366 standardized uptake value
Standard Deviation 1.1814
|
-0.432 standardized uptake value
Standard Deviation 0.5963
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 monthsPopulation: Safety population: All participants who received at least 1 dose of paxalisib
Response to treatment was assessed by MRI using the response assessment in neuro-oncology (RANO) criteria based on the assessment of the MRI scan and clinical features. The DCR is defined as the proportion of patients achieving a confirmed best overall response of complete response (CR; disappearance of all enhancing disease, clinically stable/improved), partial response (PR; 50% or more decrease in measurable enhancing lesions, clinically stable/improved), or stable disease (SD, does not qualify for CR or PR, does not qualify for disease progression, clinically stable). To be assigned a status of CR, PR or SD patients need to have two consecutive assessments of CR, PR or SD. To be assigned a best overall response of SD patients must have a minimum duration of SD of at least 6 weeks.
Outcome measures
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 Participants
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 Participants
Stage 1, Cohort 2
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 Participants
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 Participants
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
Disease Control Rate.
|
3 Participants
|
4 Participants
|
8 Participants
|
9 Participants
|
Adverse Events
Dose-escalating Cohort 1: Paxalisib 60mg
Serious events: 2 serious events
Other events: 3 other events
Deaths: 2 deaths
Dose-escalating Cohort 2: Paxalisib 75 mg
Serious events: 5 serious events
Other events: 6 other events
Deaths: 4 deaths
Expansion Cohort: Paxalisib 60mg (Fed)
Serious events: 6 serious events
Other events: 10 other events
Deaths: 8 deaths
Expansion Cohort: Paxalisib 60mg (Fasted)
Serious events: 5 serious events
Other events: 11 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 participants at risk
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 participants at risk
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 participants at risk
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 participants at risk
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
General disorders
Fatigue
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Dehydration
|
33.3%
1/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Psychiatric disorders
confusional state
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Status epilepticus
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Skin and subcutaneous tissue disorders
Drug reaction with eosinophilia and systemic symptoms
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Infections and infestations
Meningitis
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Renal and urinary disorders
Acute kidney injury
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Skin and subcutaneous tissue disorders
Dermatitis exfoliative
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Posterior reversible encephalopathy syndrome
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Central nervous system haemorrhage
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Cerebral ischaemia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Reversible cerebral vasoconstriction syndrome
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Infections and infestations
Wound infection
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
General disorders
Gait disturbance
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Vascular disorders
Embolism
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Vasogenic cerebral oedema
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Hydrocephalus
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Embolic stroke
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
Other adverse events
| Measure |
Dose-escalating Cohort 1: Paxalisib 60mg
n=3 participants at risk
Stage 1, Cohort 1
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach
|
Dose-escalating Cohort 2: Paxalisib 75 mg
n=6 participants at risk
Participants were administered 75 mg paxalisib (5 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fed)
n=10 participants at risk
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants consumed a high-fat, high-calorie meal approximately 30 minutes prior to dosing, and fasted thereafter for at least 4 hours on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
Expansion Cohort: Paxalisib 60mg (Fasted)
n=11 participants at risk
Stage 2, Fasted
Participants were administered 60 mg paxalisib (4 ×15 mg capsules) orally once per day in 28-day cycles. Participants fasted for 10 hours pre-dose and for 4 hours post-dose on days when they underwent PK assessments (Cycle 1 Day 1 and Cycle 2 Day 1). On all other days doses were administered at least 1 hour before or 2 hours after food to ensure the study medication was taken on an empty stomach.
|
|---|---|---|---|---|
|
General disorders
Fatigue
|
100.0%
3/3 • Number of events 7 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
66.7%
4/6 • Number of events 8 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
60.0%
6/10 • Number of events 10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
81.8%
9/11 • Number of events 13 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
General disorders
Chills
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
18.2%
2/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
General disorders
Gait disturbance
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
General disorders
Pyrexia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
General disorders
Malaise
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
18.2%
2/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Stomatitis
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
50.0%
3/6 • Number of events 8 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
60.0%
6/10 • Number of events 10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
36.4%
4/11 • Number of events 5 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Nausea
|
66.7%
2/3 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
40.0%
4/10 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
36.4%
4/11 • Number of events 6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
50.0%
3/6 • Number of events 7 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
45.5%
5/11 • Number of events 6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Vomiting
|
66.7%
2/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
27.3%
3/11 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Dysphagia
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Gastrointestinal disorders
Constipation
|
66.7%
2/3 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
50.0%
5/10 • Number of events 7 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
54.5%
6/11 • Number of events 8 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
33.3%
1/3 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
83.3%
5/6 • Number of events 10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
30.0%
3/10 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
36.4%
4/11 • Number of events 10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Dehydration
|
66.7%
2/3 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
40.0%
4/10 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
18.2%
2/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 5 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Metabolism and nutrition disorders
Hypertrigliceridaemia
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
33.3%
1/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
45.5%
5/11 • Number of events 8 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 5 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
36.4%
4/11 • Number of events 6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Headache
|
33.3%
1/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Aphasia
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
50.0%
3/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
18.2%
2/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Dysgeusia
|
33.3%
1/3 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
18.2%
2/11 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Cognitive disorder
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Nervous system disorders
Seizure
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Platelet count decreased
|
66.7%
2/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
66.7%
4/6 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Neutrophil count decreased
|
33.3%
1/3 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
27.3%
3/11 • Number of events 5 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Weight decreased
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
30.0%
3/10 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Lymphocyte count decreased
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
50.0%
3/6 • Number of events 12 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Blood cholesterol increased
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/6 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Investigations
Blood creatinine increased
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
16.7%
1/6 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Infections and infestations
Urinary tract infection
|
33.3%
1/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
20.0%
2/10 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Psychiatric disorders
Depression
|
33.3%
1/3 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Vascular disorders
Hypertension
|
33.3%
1/3 • Number of events 4 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/10 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
9.1%
1/11 • Number of events 2 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
|
Blood and lymphatic system disorders
Anaemia
|
33.3%
1/3 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
33.3%
2/6 • Number of events 3 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
10.0%
1/10 • Number of events 1 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
0.00%
0/11 • 24 months
Adverse events may have been volunteered spontaneously by the patient, discovered as a result of general questioning by the study staff, or determined by physical examination. Adverse events were followed until they resolved, were stable, or until the patient's last study visit or were lost to follow-up.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60