Trial Outcomes & Findings for RAD001 in Advanced Hepatocellular Carcinoma (NCT NCT00516165)
NCT ID: NCT00516165
Last Updated: 2017-02-07
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
28 participants
Primary outcome timeframe
2 years
Results posted on
2017-02-07
Participant Flow
Participant milestones
| Measure |
RAD001
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Overall Study
STARTED
|
28
|
|
Overall Study
COMPLETED
|
28
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
RAD001 in Advanced Hepatocellular Carcinoma
Baseline characteristics by cohort
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Age, Continuous
|
65 years
n=99 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=99 Participants
|
|
Gender
Female
|
10 Participants
n=99 Participants
|
|
Gender
Male
|
18 Participants
n=99 Participants
|
|
Region of Enrollment
United States
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28 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 2 yearsPopulation: Patients with histologically confirmed measurable advanced Hepatocellular Carcinoma.
Outcome measures
| Measure |
RAD001
n=25 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Maximum Tolerated Dose of RAD001 in Patients With Advanced Hepatocellular Carcinoma (HCC).
|
10 mg
|
PRIMARY outcome
Timeframe: 2 yearsProgression was defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions" This information will be collected during two years of patient participation.
Outcome measures
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Progression-free Survival Rate at 24 Weeks
|
3.8 months
Interval 2.1 to 4.6
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SECONDARY outcome
Timeframe: 2 yearsPopulation: Patients with histologically confirmed measurable advanced HCC. The primary end points were determination of a safe dosage of everolimus and progression-free survival at 24 weeks.
Everolimus given at 10 mg/day as a single agent was well tolerated in patients with advanced HCC.
Outcome measures
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Number of Patients With Adverse Events Who Were Treated With RAD001 for Advanced HCC
|
28 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPer Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Outcome measures
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Overall Response Rate
|
4 percentage of patient response
Interval 0.9 to 19.6
|
SECONDARY outcome
Timeframe: 2 years3.9 months with a CI of 21-
Outcome measures
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Time to Progression
|
3.9 month
Interval 2.1 to 5.5
|
SECONDARY outcome
Timeframe: 2 yearsThe median overall survival was 8.4 months (95% CI, 3.9-21.1 months). Only 2 ((8 %) patients were progression-free at 24 weeks. The study did not proceed to the second stage of the phase 2 portion of the study.
Outcome measures
| Measure |
RAD001
n=28 Participants
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
Overall Survival
|
8.4 months
Interval 3.9 to 21.1
|
Adverse Events
RAD001
Serious events: 7 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
RAD001
n=28 participants at risk
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
General disorders
Grade 5-Death within 30 days.
|
14.3%
4/28 • Number of events 4 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Nervous system disorders
Grade 3 Encephalopathy-unrelated
|
3.6%
1/28 • Number of events 1 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Metabolism and nutrition disorders
Grade 4 Anemia
|
3.6%
1/28 • Number of events 1 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Metabolism and nutrition disorders
Grade 4 Thrombocytopenia
|
3.6%
1/28 • Number of events 1 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
General disorders
Grade 4-Hypoxia
|
3.6%
1/28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Grade 4 Bilirubin
|
3.6%
1/28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Blood and lymphatic system disorders
Grade 4 Hypophosphatemis
|
3.6%
1/28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
General disorders
Respiratory failure
|
3.6%
1/28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Blood and lymphatic system disorders
Grade 4 elevated SGOT
|
3.6%
1/28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
Other adverse events
| Measure |
RAD001
n=28 participants at risk
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
RAD001: Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
|
|---|---|
|
General disorders
Fatigue
|
46.4%
13/28 • Number of events 52 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
42.9%
12/28 • Number of events 48 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Blood and lymphatic system disorders
Anemia
|
39.3%
11/28 • Number of events 44 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Diarrhea
|
39.3%
11/28 • Number of events 44 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Leukopenia
|
35.7%
10/28 • Number of events 40 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Platelets
|
35.7%
10/28 • Number of events 40 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Aspartate transaminase
|
32.1%
9/28 • Number of events 36 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Metabolism and nutrition disorders
Hyponatremia
|
32.1%
9/28 • Number of events 36 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Metabolism and nutrition disorders
Anorexia
|
32.1%
9/28 • Number of events 36 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Blood and lymphatic system disorders
Lymphopenia
|
28.6%
8/28 • Number of events 32 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Stomatitis
|
25.0%
7/28 • Number of events 28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
7/28 • Number of events 28 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Alanine transaminase
|
21.4%
6/28 • Number of events 24 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Neutropenia
|
17.9%
5/28 • Number of events 20 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Alkaline phosphatase
|
14.3%
4/28 • Number of events 16 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Proteinuria
|
14.3%
4/28 • Number of events 16 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
4/28 • Number of events 16 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Nausea
|
14.3%
4/28 • Number of events 16 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
14.3%
4/28 • Number of events 16 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Weight loss
|
10.7%
3/28 • Number of events 12 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Skin and subcutaneous tissue disorders
Acne rash
|
10.7%
3/28 • Number of events 12 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Immune system disorders
Edema
|
10.7%
3/28 • Number of events 12 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Nervous system disorders
Taste disturbance
|
10.7%
3/28 • Number of events 12 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Hypoalbuminemia
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Hypokalemia
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Hypertriglyceridemia
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Fever without neuropenia
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Constipation
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Investigations
Nose bleeds
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
General disorders
Cough
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
General disorders
Insomnia
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
|
Gastrointestinal disorders
Pain
|
7.1%
2/28 • Number of events 8 • October 2007 and June 2009; Participants were followed for this two year time period.
28 patients entered into the trial (9 in phase 1, 19 in phase 2) and all were evaluable for efficacy and toxicity based on intention-to-treat analysis. There were 18 men (64%) and 10 women (36%) with a median age of 65 years (range, 33-81 years).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place