Trial Outcomes & Findings for A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome (NCT NCT05098509)
NCT ID: NCT05098509
Last Updated: 2023-10-19
Results Overview
The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Recruitment status
TERMINATED
Study phase
PHASE2/PHASE3
Target enrollment
4 participants
Primary outcome timeframe
Baseline, Week 34
Results posted on
2023-10-19
Participant Flow
In this study, 4 participants were randomized and received at least 1 dose of study drug. No participants were randomized to the placebo group. The 4 participants were randomized in Phase 2 of the study and due to premature termination of the study, phase 3 was not conducted. None of the enrolled participants completed the study due to the study being terminated prematurely.
Participant milestones
| Measure |
RAD011 10 Milligrams Per Kilogram (mg/kg)
Participants were administered 10 mg/kg of RAD011 orally daily with food.
|
RAD011 20 mg/kg
Participants were administered 20 mg/kg of RAD011 orally daily with food.
|
RAD011 40 mg/kg
Participants were administered 40 mg/kg of RAD011 orally daily with food.
|
Placebo
Participants were administered a placebo matching to RAD011, orally daily with food.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
2
|
0
|
|
Overall Study
Received 1 Dose of Study Drug
|
1
|
1
|
2
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
2
|
0
|
Reasons for withdrawal
| Measure |
RAD011 10 Milligrams Per Kilogram (mg/kg)
Participants were administered 10 mg/kg of RAD011 orally daily with food.
|
RAD011 20 mg/kg
Participants were administered 20 mg/kg of RAD011 orally daily with food.
|
RAD011 40 mg/kg
Participants were administered 40 mg/kg of RAD011 orally daily with food.
|
Placebo
Participants were administered a placebo matching to RAD011, orally daily with food.
|
|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
1
|
0
|
|
Overall Study
Early study termination
|
1
|
1
|
1
|
0
|
Baseline Characteristics
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
Baseline characteristics by cohort
| Measure |
RAD011 10 mg/kg
n=1 Participants
Participants were administered 10 mg/kg of RAD011 orally daily with food.
|
RAD011 20 mg/kg
n=1 Participants
Participants were administered 20 mg/kg of RAD011 orally daily with food.
|
RAD011 40 mg/kg
n=2 Participants
Participants were administered 40 mg/kg of RAD011 orally daily with food.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
NA years
n=1 Participants
|
NA years
n=1 Participants
|
NA years
n=2 Participants
|
NA years
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Sex: Female, Male
Male
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
Asian
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
White
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
0 Participants
Due to the small sample size for this baseline characteristic, data was not reported for confidentiality reasons.
|
PRIMARY outcome
Timeframe: Baseline, Week 34Population: Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure.
The HQ-CT measures hyperphagia by Prader-Willi syndrome (PWS)-specialized clinicians. The HQ-CT generates a score ranging from 0 to 36, where a higher score represents more severe abnormal food related behaviors. The change from baseline was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 34Population: Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure.
The ABC questionnaire is an informant-rated questionnaire assessing severity of behavioral symptoms. The Irritability subscale of the ABC covers symptoms such as agitation, aggression, meltdowns, and self-harm. The ABC-I contains 15 items and each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score is the sum of individual items scores which ranges from 0 (no problem) to 45 (severe problem), with higher score indicating more severe condition.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 34Population: Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure.
The CGI-C of hyperphagia is a single-item, clinician-rated measure, assessing the clinician's impression about changes in the patient's hyperphagia condition since the start of taking the study medication at the initiation of the Tolerability Period. The CGI-C of hyperphagia utilizes a 5-point response scale: 1=Much better; 2=A little better; 3=No change; 4=A little worse; 5=Much worse. Higher scores mean a worse outcome.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Week 34Population: Due to the premature termination of the study, no efficacy analyses were conducted. Therefore, no data was collected for this outcome measure.
The CGI-S of hyperphagia is a single-item, clinician-rated measure, assessing the clinician's impression of the severity of a patient's hyperphagia condition. The CGI-S of hyperphagia utilizes a 5-point response scale: 1=None; 2=Mild; 3=Moderate; 4=Severe; 5=Very Severe. Higher scores mean a worse outcome.
Outcome measures
Outcome data not reported
Adverse Events
RAD011 10 mg/kg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
RAD011 20 mg/kg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
RAD011 40 mg/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RAD011 10 mg/kg
n=1 participants at risk
Participants were administered 10 mg/kg of RAD011 orally daily with food.
|
RAD011 20 mg/kg
n=1 participants at risk
Participants were administered 20 mg/kg of RAD011 orally daily with food.
|
RAD011 40 mg/kg
n=2 participants at risk
Participants were administered 40 mg/kg of RAD011 orally daily with food.
|
|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
100.0%
1/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
100.0%
1/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
100.0%
2/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
Psychiatric disorders
Irritability
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
50.0%
1/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
Nervous system disorders
Headache
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
50.0%
1/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
General disorders
Fatigue
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
50.0%
1/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
|
Ear and labyrinth disorders
Ear discomfort
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
0.00%
0/1 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
50.0%
1/2 • Day 1 (after dosing) up to 68 days (maximum duration from treatment to early termination)
Safety population included all participants who received at least 1 dose of study medication.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place