Trial Outcomes & Findings for Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome (NCT NCT02844933)

Last Updated: 2023-08-01

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: value at Week 13 minus value at Baseline.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

7 participants

Primary outcome timeframe

Baseline, Week 13

Results posted on

2023-08-01

Participant Flow

Participants took part in the study at 5 investigative sites in the United States from 09 May 2018 and 31 July 2019.

Approximately 66 participants aged 8 to 17 years, inclusive, with a genetically confirmed diagnosis of Prader-Willi Syndrome were planned to be enrolled in the study. The previous Sponsor prematurely terminated the study prior to reaching the planned enrollment. Therefore, only 7 participants participated in the study.

Participant milestones

Participant milestones
Measure	Cannabidiol Participants received cannabidiol oral solution of 40 milligram/kilogram/day (mg/kg/day) divided into two daily doses with a standard meal.	Placebo Participants received a matching placebo solution divided into two daily doses with a standard meal.
Overall Study STARTED	4	3
Overall Study Received at Least 1 Dose of Study Drug	4	3
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	3	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Cannabidiol Participants received cannabidiol oral solution of 40 milligram/kilogram/day (mg/kg/day) divided into two daily doses with a standard meal.	Placebo Participants received a matching placebo solution divided into two daily doses with a standard meal.
Overall Study Discontinued due to premature termination of study	3	2

Baseline Characteristics

Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cannabidiol n=4 Participants Participants received cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal.	Placebo n=3 Participants Participants received matching placebo solution divided into two daily doses with a standard meal.	Total n=7 Participants Total of all reporting groups
Age, Continuous	13.5 years n=5 Participants	11.67 years n=7 Participants	12.7 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Baseline in Total Body Weight	88.35 kg n=5 Participants	56.7 kg n=7 Participants	72.50 kg n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 13

Population: All participants who received at least 1 dose of study drug

Outcome measures

Outcome measures
Measure	Cannabidiol n=4 Participants Participants received cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal.	Placebo n=3 Participants Participants received matching placebo solution divided into two daily doses with a standard meal.
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Baseline	23 score on a scale Interval 10.0 to 25.0	22 score on a scale Interval 18.0 to 26.0
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Week 13	14 score on a scale Interval 7.0 to 20.0	16 score on a scale Interval 10.0 to 28.0
Change From Baseline in Hyperphagia Behavior as Measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Change from Baseline at Week 13	-4.5 score on a scale Interval -14.0 to -3.0	-2 score on a scale Interval -12.0 to 2.0

SECONDARY outcome

Timeframe: Baseline, Week 13

Population: All participants who received at least 1 dose of study drug

Total body weight refers to participants' weight throughout the study. 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: value at Week 13 minus value at Baseline.

Outcome measures

Outcome measures
Measure	Cannabidiol n=4 Participants Participants received cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal.	Placebo n=3 Participants Participants received matching placebo solution divided into two daily doses with a standard meal.
Change From Baseline In Total Body Weight Baseline	88.35 kg Interval 47.9 to 112.4	56.7 kg Interval 45.5 to 109.7
Change From Baseline In Total Body Weight Week 13	87.6 kg Interval 47.4 to 110.8	59.5 kg Interval 46.8 to 109.4
Change From Baseline In Total Body Weight Change From Baseline at Week 13	-1.05 kg Interval -1.6 to 0.1	1.3 kg Interval -0.3 to 1.3

SECONDARY outcome

Timeframe: Baseline up to Week 13

Population: All participants who received at least 1 dose of study drug

A responder was defined as having a 6 or more points decrease in HQ-CT score.

Outcome measures

Outcome measures
Measure	Cannabidiol n=4 Participants Participants received cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal.	Placebo n=3 Participants Participants received matching placebo solution divided into two daily doses with a standard meal.
Responder Rate From Baseline Through Study Completion Yes	1 Participants	1 Participants
Responder Rate From Baseline Through Study Completion No	3 Participants	2 Participants

SECONDARY outcome

Timeframe: Week 3, Week 13

Population: Due to early study termination, data was not collected for analysis of this outcome measure.

The change in PGI-C scores at Week 3, Week 9, and Week 13 of the participant are evaluated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 13

Population: Due to early study termination, data was not collected for analysis of this outcome measure.

The change from baseline to Week 13 of the participant's TFEQ-R18 score is evaluated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 13

Population: Due to early study termination, data was not collected for analysis of this outcome measure.

The change from baseline to Week 13 of the participant's quality of life (PROMIS life satisfaction and positive affect questionnaire) score is evaluated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 13

Population: Due to early study termination, data was not collected for analysis of this outcome measure.

The change from baseline to Week 13 of the participant's physical activity (PROMIS physical activity and fatigue questionnaires) score is evaluated.

Outcome measures

Outcome data not reported

Adverse Events

Cannabidiol

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cannabidiol n=4 participants at risk Participants received cannabidiol oral solution (40 mg/kg/day) divided into two daily doses with a standard meal.	Placebo n=3 participants at risk Participants received matching placebo solution divided into two daily doses with a standard meal.
Gastrointestinal disorders Diarrhea	50.0% 2/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	33.3% 1/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Skin and subcutaneous tissue disorders Rash	50.0% 2/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Gastrointestinal disorders Abdominal cramping	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Infections and infestations Upper respiratory tract infection	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	33.3% 1/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Nervous system disorders Seizure	0.00% 0/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	33.3% 1/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Infections and infestations Impetigo	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Infections and infestations Cellulitis	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Ear and labyrinth disorders Ear infection	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.
Infections and infestations Bacterial infection	25.0% 1/4 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.	0.00% 0/3 • Baseline up to Week 13 Safety population included all participants who received at least 1 dose of study medication.

Additional Information

Tarek El Akkad, Head of Clinical Development

Radius Pharmaceuticals, Inc.

Phone: 6175514000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60