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A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome

Primary Purpose

Prader-Willi Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
RAD011
Placebo
Sponsored by
Radius Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome

Eligibility Criteria

8 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Males and females between 8 and 65 years of age (inclusive) at Screening
  • Genetically confirmed diagnosis of PWS. Documentation of genetically confirmed diagnosis of PWS is acceptable.
  • The same caregiver is available to complete the questionnaire throughout the duration of the study.
  • After completion of the Tolerability period, Patients will have a mean HQ-CT score >= 13 and a decrease of HQ-CT score no more than 7 during Tolerability (run-in) period
  • If receiving growth hormone, psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and dose for at least 90 days prior to consent/assent

Exclusion Criteria:

  • Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/ assent
  • Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent
  • Implementation of new food or environmental restrictions within 90 days of consent/ assent
  • If living in a group home, patient spends less than 25 waking hours with their caregiver per week
  • Uncontrolled chronic conditions (diabetes, sleep apnea, etc.) as determined by the Investigator

Sites / Locations

  • Children's Hospital Los Angeles
  • Rady Children's Hospital
  • University of Iowa
  • Maimonides Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Vanderbilt University Medical Center
  • Research Institute of Dallas
  • MultiCare Institute for Research & Innovation

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Active Comparator

Active Comparator

Active Comparator

Placebo Comparator

Arm Label

RAD011 40 milligrams per kilogram (mg/kg)

RAD011 20 mg/kg

RAD011 10 mg/kg

Placebo

Arm Description

Participants were administered 40 mg/kg of RAD011 orally daily with food.

Participants were administered 20 mg/kg of RAD011 orally daily with food.

Participants were administered 10 mg/kg of RAD011 orally daily with food.

Participants were administered a placebo matching to RAD011, orally daily with food.

Outcomes

Primary Outcome Measures

Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
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.

Secondary Outcome Measures

Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)
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.
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)
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.
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)
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.

Full Information

First Posted
September 22, 2021
Last Updated
September 26, 2023
Sponsor
Radius Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05098509
Brief Title
A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome
Official Title
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader- Willi Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Terminated
Why Stopped
Due to a change in corporate priorities, this study was voluntarily terminated by the Sponsor for reasons other than safety
Study Start Date
April 13, 2022 (Actual)
Primary Completion Date
October 6, 2022 (Actual)
Study Completion Date
October 31, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Radius Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This was a study investigating RAD011 in participants diagnosed with Prader-Willi Syndrome (PWS). The primary objective of the Phase 2 part of this study was to assess the safety and tolerability of multiple dose levels of RAD011 in order to select 1 or 2 dose level(s) for further evaluation in the Phase 3 part of the study. In Phase 3, the primary objective was to assess the effect of RAD011 on hyperphagia-related behavior in participants with PWS.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
RAD011 40 milligrams per kilogram (mg/kg)
Arm Type
Active Comparator
Arm Description
Participants were administered 40 mg/kg of RAD011 orally daily with food.
Arm Title
RAD011 20 mg/kg
Arm Type
Active Comparator
Arm Description
Participants were administered 20 mg/kg of RAD011 orally daily with food.
Arm Title
RAD011 10 mg/kg
Arm Type
Active Comparator
Arm Description
Participants were administered 10 mg/kg of RAD011 orally daily with food.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants were administered a placebo matching to RAD011, orally daily with food.
Intervention Type
Drug
Intervention Name(s)
RAD011
Intervention Description
Cannabidiol Oral Solution (containing synthetic cannabidiol)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Matching Placebo for RAD011
Primary Outcome Measure Information:
Title
Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
Description
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.
Time Frame
Baseline, Week 34
Secondary Outcome Measure Information:
Title
Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)
Description
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.
Time Frame
Baseline, Week 34
Title
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)
Description
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.
Time Frame
Baseline, Week 34
Title
Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)
Description
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.
Time Frame
Baseline, Week 34

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Males and females between 8 and 65 years of age (inclusive) at Screening. Genetically confirmed diagnosis of PWS. Documentation of genetically confirmed diagnosis of PWS is acceptable. The same caregiver is available to complete the questionnaire throughout the duration of the study. After completion of the Tolerability period, participants will have a mean Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score ≥13 and a decrease of HQ-CT score no more than 7 during Tolerability (run-in) period. If receiving growth hormone, psychotropic therapy, metabolic treatments that could affect appetite (including metformin), and other treatment including thyroid hormone, must be on the same medication and dose for at least 90 days prior to consent/assent Exclusion Criteria: Known use of cannabis or cannabinoid containing products (including topical products) within 90 days prior to consent/ assent. Use of prescription or over-the-counter weight loss agents within 90 days prior to consent/assent. Implementation of new food or environmental restrictions within 90 days of consent/ assent. If living in a group home, participant spends less than 25 waking hours with their caregiver per week. Uncontrolled chronic conditions (diabetes, sleep apnea, etc.) as determined by the Investigator.
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Rady Children's Hospital
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Maimonides Medical Center
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11219
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Facility Name
Research Institute of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
MultiCare Institute for Research & Innovation
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States

12. IPD Sharing Statement

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A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome

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