Trial Outcomes & Findings for Epidiolex (CBD) in Patients With Biochemically Recurrent Prostate Cancer (NCT NCT04428203)
NCT ID: NCT04428203
Last Updated: 2025-03-05
Results Overview
DLT was defined as grade ≥3 nausea, vomiting, diarrhea that persists \>72 h despite optimal anti-emetics and anti-diarrhea treatment, grade ≥3 hematological adverse events (AEs), or grade ≥2 suicidal ideation.Treatment-related adverse events are those that comprise a dose-limiting toxicity within 30 days after initiation of Epidiolex (i.e., acute DLT). Additionally, Treatment-related adverse events will continue to be monitored for a total of 90 days.
COMPLETED
PHASE1
21 participants
up to 90 days
2025-03-05
Participant Flow
Participant milestones
| Measure |
Experimental: Dose Escalation: Epidiolex 600 mg
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Experimental: Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Experimental: Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
3
|
14
|
|
Overall Study
COMPLETED
|
2
|
3
|
13
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
1
|
Reasons for withdrawal
| Measure |
Experimental: Dose Escalation: Epidiolex 600 mg
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Experimental: Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Experimental: Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
1
|
Baseline Characteristics
Epidiolex (CBD) in Patients With Biochemically Recurrent Prostate Cancer
Baseline characteristics by cohort
| Measure |
Dose Escalation: Epidiolex 600 mg
n=4 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation and Dose Expansion: Epidiolex 800 mg
n=17 Participants
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
68 years
n=99 Participants
|
71 years
n=107 Participants
|
69 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 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
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=99 Participants
|
17 participants
n=107 Participants
|
21 participants
n=206 Participants
|
|
PSA (ng/ml)
|
1.84 ng/ml
n=99 Participants
|
1.25 ng/ml
n=107 Participants
|
1.25 ng/ml
n=206 Participants
|
|
Testosterone, total (ng/dL)
|
214.5 mg/dL
n=99 Participants
|
360 mg/dL
n=107 Participants
|
325 mg/dL
n=206 Participants
|
|
Prior treatment
Prostatectomy Alone
|
1 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Prior treatment
Radiation Alone
|
1 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Prior treatment
Prostatectomy and salvage radiation
|
2 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Prior treatment
Untreated
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: up to 90 daysPopulation: Among 21 patients, a total of 18 patients were included in the efficacy analysis
DLT was defined as grade ≥3 nausea, vomiting, diarrhea that persists \>72 h despite optimal anti-emetics and anti-diarrhea treatment, grade ≥3 hematological adverse events (AEs), or grade ≥2 suicidal ideation.Treatment-related adverse events are those that comprise a dose-limiting toxicity within 30 days after initiation of Epidiolex (i.e., acute DLT). Additionally, Treatment-related adverse events will continue to be monitored for a total of 90 days.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=2 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
n=3 Participants
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
n=13 Participants
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Number of Participants With Dose-limiting Toxicities (Treatment-related Adverse Events) as Assessed by the CTCAE v5.0
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: within 90 daysPopulation: Patients who were DLT evaluable, had baseline measurements, and had at least one post-baseline measurement were included in the analysis of secondary endpoints. The assessments were combined for patients who received the recommended dose of 800 mg daily.
Biochemical response (25% change from baseline) will be determined by the measurement of PSA at baseline and approximately every 4 weeks during treatment.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=2 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
n=16 Participants
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Participants With Biochemical Response.
stable biochemical disease
|
2 Participants
|
14 Participants
|
—
|
|
Participants With Biochemical Response.
partial biochemical response
|
0 Participants
|
1 Participants
|
—
|
|
Participants With Biochemical Response.
PSA progression
|
0 Participants
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: within 90 daysPopulation: Patients who were DLT evaluable, had baseline measurements, and had at least one post-baseline measurement were included in the analysis of secondary endpoints. The assessments were combined for patients who received the recommended dose of 800 mg daily.
Biochemical response will be determined by measurement of PSA approximately every 4 weeks during treatment. PSA velocity is the calculation (PSA final measurement - PSA initial measurement) / number of years between measurements.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=16 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
n=2 Participants
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Change in PSA Velocity From Baseline Throughout the Treatment Period as an Indication of Biochemical Response.
|
0 nanograms per milliliter per year
Standard Deviation 0
|
0 nanograms per milliliter per year
Standard Deviation 0
|
—
|
SECONDARY outcome
Timeframe: Baseline, Day 1 of Cycle 1 (each cycle is 4 weeks), Day 1 of Cycle 2, Day 1 of Cycle 3, and 1 month post treatment (up to 16 weeks)Population: Patients who were DLT evaluable, had baseline measurements, and had at least one post-baseline measurement were included in the analysis of secondary endpoints. Data analysis for this outcome was combined in the 800 mg group
Biochemical response will be determined by measurement of total testosterone level approximately every 4 weeks during treatment.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=16 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
n=2 Participants
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response
Cycle 2
|
345.21 ng/dL
Standard Deviation 157.02
|
242 ng/dL
Standard Deviation 14.14
|
—
|
|
Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response
Cycle 3
|
253.92 ng/dL
Standard Deviation 249.61
|
208 ng/dL
Standard Deviation 36.77
|
—
|
|
Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response
Baseline
|
388.3 ng/dL
Standard Deviation 270.7
|
214.5 ng/dL
Standard Deviation 30.4
|
—
|
|
Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response
Cycle 1
|
421.94 ng/dL
Standard Deviation 238.67
|
190 ng/dL
Standard Deviation 76.37
|
—
|
|
Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response
1 month post treatment
|
434.0 ng/dL
Standard Deviation 252.8
|
190.0 ng/dL
Standard Deviation 76.4
|
—
|
SECONDARY outcome
Timeframe: BaselinePopulation: No data released for 600mg as only 1 participant answered QOL measures and confidentiality of health information could be impacted if the outcome measures were displayed. Only patients who received one or more doses of the recommended phase 2 dose (800 mg) and who completed one or more sections of the quality-of-life assessment were analyzed. Of the 17 patients who received the recommended dose of 800 mg daily, 17 completed patient-reported outcomes at baseline and 12 completed PROs at 12 weeks.
The EORTC quality of life questionnaire (QLQ) 30 is a validated 30-item patient-reported questionnaire assessing quality of life among cancer populations. The quality of life questionnaire-C30 is the core QOL instrument, with 30 items that comprise five functioning scales (physical, social, role, cognitive, and emotional functioning), eight symptom scales (fatigue, nausea/vomiting, pain, dyspnea, sleep disturbances, appetite loss, constipation, and diarrhea), financial impact, and overall quality of life. All raw item scores are transformed to scale scores, linearly converted to range from 0 to 100. For the functioning scales and global QOL, higher scores indicate better functioning. For the symptom scales, higher scores indicate higher symptom burden.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=17 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Dyspnea
|
7.8 units on a scale
Standard Deviation 14.6
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Physical Functioning
|
92.9 units on a scale
Standard Deviation 11.9
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Role Functioning
|
97.1 units on a scale
Standard Deviation 8.8
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Emotional Functioning
|
94.1 units on a scale
Standard Deviation 9.2
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Cognitive Functioning
|
93.1 units on a scale
Standard Deviation 10.3
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Insomnia
|
13.7 units on a scale
Standard Deviation 16.9
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Appetite Loss
|
0.0 units on a scale
Standard Deviation 0.0
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Social Funcitoning
|
97.1 units on a scale
Standard Deviation 8.8
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Fatigue
|
12.4 units on a scale
Standard Deviation 13.0
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Constipation
|
3.9 units on a scale
Standard Deviation 11.1
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Diarrhea
|
7.8 units on a scale
Standard Deviation 14.6
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Financial Problems
|
7.8 units on a scale
Standard Deviation 14.6
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Nausea/Vomiting
|
1.0 units on a scale
Standard Deviation 4.0
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Pain
|
15.7 units on a scale
Standard Deviation 21.6
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Global Health Status/QOL
|
78.4 units on a scale
Standard Deviation 15.0
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: No data released for 600mg as only 1 participant answered QOL measures and confidentiality of health information could be impacted if the outcome measures were displayed. Only patients who received one or more doses of the recommended phase 2 dose (800 mg) and who completed one or more sections of the quality-of-life assessment were analyzed. Of the 17 patients who received the recommended dose of 800 mg daily, 17 completed patient-reported outcomes at baseline and 12 completed PROs at 12 weeks.
The EORTC quality of life questionnaire (QLQ) 30 is a validated 30-item patient-reported questionnaire assessing quality of life among cancer populations. The quality of life questionnaire-C30 is the core QOL instrument, with 30 items that comprise five functioning scales (physical, social, role, cognitive, and emotional functioning), eight symptom scales (fatigue, nausea/vomiting, pain, dyspnea, sleep disturbances, appetite loss, constipation, and diarrhea), financial impact, and overall quality of life. All raw item scores are transformed to scale scores, linearly converted to range from 0 to 100. For the functioning scales and global QOL, higher scores indicate better functioning. For the symptom scales, higher scores indicate higher symptom burden.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=12 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Physical Functioning
|
90.0 units on a scale
Standard Deviation 14.4
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Diarrhea
|
8.3 units on a scale
Standard Deviation 15.1
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Global Health Status/QOL
|
81.3 units on a scale
Standard Deviation 13.4
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Role Functioning
|
93.1 units on a scale
Standard Deviation 13.2
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Emotional Functioning
|
96.5 units on a scale
Standard Deviation 5.6
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Cognitive Functioning
|
91.7 units on a scale
Standard Deviation 11.2
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Social Funcitoning
|
95.8 units on a scale
Standard Deviation 10.4
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Fatigue
|
16.7 units on a scale
Standard Deviation 18.7
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Nausea/Vomiting
|
1.4 units on a scale
Standard Deviation 4.8
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Pain
|
13.9 units on a scale
Standard Deviation 17.2
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Dyspnea
|
13.9 units on a scale
Standard Deviation 22.3
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Insomnia
|
8.3 units on a scale
Standard Deviation 20.7
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Appetite Loss
|
0.0 units on a scale
Standard Deviation 0.0
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Constipation
|
13.9 units on a scale
Standard Deviation 17.2
|
—
|
—
|
|
Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)
Financial Problems
|
11.1 units on a scale
Standard Deviation 16.4
|
—
|
—
|
SECONDARY outcome
Timeframe: BaselinePopulation: Assessments were combined and analyzed for patients who received one or more doses of the recommended phase 2 dose and who completed one or more sections of the quality-of-life assessment. For 800mg dose17 completed baseline and 12 completed at 12 weeks. Some participants left some questions blank (incontinence, sexual activity). No data released for 600mg as only 1 participant answered QOL measures, confidentiality of health information could be impacted if the outcome measures were displayed.
The EORTC quality of life questionnaire (QLQ)-PR25 is a validated 25-item patient-reported questionnaire which complements the EORTC QLQ-C30,core QOL questionnaire. The QLQ-PR25 comprises 25 items assessing sequelae specific to prostate cancer and its treatment, and thus, is intended to supplement the EORTC QLQ-C30. The 25 items comprise six prostate-specific scales: Urinary, Bowel, Use of Incontinence Aids, Prostate Cancer Treatment-Related Symptoms, Sexual Active and Sexual Function. Raw item scores are linearly transformed to a 0 to 100 scale (i.e., same unit of measurement used by the core QLQ-C30 questionnaire). For the QLQ-PR25, higher scores on symptom domains (e.g., urinary, bowel, etc.) indicate greater symptom burden. Higher scores on function domains (e.g., Sexual Function) indicate better functioning.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=17 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Bowel Symptoms
|
6.4 units on a scale
Standard Deviation 8.1
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Urinary Symptoms
|
17.9 units on a scale
Standard Deviation 12.7
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Hormonal Treatment Related Symptoms
|
5.9 units on a scale
Standard Deviation 6.0
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Sexual activity
|
67.6 units on a scale
Standard Deviation 22.3
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Sexual Functioning
|
56.9 units on a scale
Standard Deviation 26.8
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Incontinence aid
|
9.5 units on a scale
Standard Deviation 16.3
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Assessments were combined and analyzed for patients who received one or more doses of the recommended phase 2 dose and who completed one or more sections of the quality-of-life assessment. For 800mg dose17 completed baseline and 12 completed at 12 weeks. Some participants left some questions blank (incontinence, sexual activity). No data released for 600mg as only 1 participant answered QOL measures, confidentiality of health information could be impacted if the outcome measures were displayed.
The EORTC quality of life questionnaire (QLQ)-PR25 is a validated 25-item patient-reported questionnaire which complements the EORTC QLQ-C30,core QOL questionnaire. The QLQ-PR25 comprises 25 items assessing sequelae specific to prostate cancer and its treatment, and thus, is intended to supplement the EORTC QLQ-C30. The 25 items comprise six prostate-specific scales: Urinary, Bowel, Use of Incontinence Aids, Prostate Cancer Treatment-Related Symptoms, Sexual Active and Sexual Function. Raw item scores are linearly transformed to a 0 to 100 scale (i.e., same unit of measurement used by the core QLQ-C30 questionnaire). For the QLQ-PR25, higher scores on symptom domains (e.g., urinary, bowel, etc.) indicate greater symptom burden. Higher scores on function domains (e.g., Sexual Function) indicate better functioning.
Outcome measures
| Measure |
Dose Escalation: Epidiolex 600 mg
n=12 Participants
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Escalation: Epidiolex 800 mg
Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Dose Expansion: Epidiolex 800 mg
Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|---|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Urinary Symptoms
|
17.5 units on a scale
Standard Deviation 11.2
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Incontinence aid
|
22.2 units on a scale
Standard Deviation 19.2
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Bowel Symptoms
|
7.6 units on a scale
Standard Deviation 8.3
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Hormonal Treatment Related Symptoms
|
9.0 units on a scale
Standard Deviation 10.2
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Sexual activity
|
63.9 units on a scale
Standard Deviation 21.1
|
—
|
—
|
|
Prostate Cancer-Specific Quality of Life (EORTC Quality of Life Questionnaire-PR25)
Sexual Functioning
|
77.8 units on a scale
Standard Deviation 0.0
|
—
|
—
|
Adverse Events
Experimental: Dose Escalation: Epidiolex 600 mg
Experimental: Dose Escalation and Expansion: Epidiolex 800 mg
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Experimental: Dose Escalation: Epidiolex 600 mg
n=2 participants at risk
Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex
|
Experimental: Dose Escalation and Expansion: Epidiolex 800 mg
n=17 participants at risk
Both Escalation and Expansion Cohorts participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
2/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
52.9%
9/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Gastrointestinal disorders
Nausea
|
100.0%
2/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
17.6%
3/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Gastrointestinal disorders
Bloating
|
0.00%
0/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Gastrointestinal disorders
Stomach Pain
|
0.00%
0/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Gastrointestinal disorders
Abnormal Liver Function Test
|
0.00%
0/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
General disorders
Fatigue
|
100.0%
2/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
11.8%
2/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
General disorders
Malaise
|
0.00%
0/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Nervous system disorders
Headache
|
50.0%
1/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Nervous system disorders
Insomnia
|
0.00%
0/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
5.9%
1/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
0.00%
0/17 • 120 days
Toxicities were graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place