A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome (SCOUT-015)

A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader- Willi Syndrome

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.

Study Overview

• Status: Terminated
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: RAD011; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
    • San Diego, California, United States, 92123
      • Rady Children's Hospital
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • New York
    • Brooklyn, New York, United States, 11219
      • Maimonides Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center
  • Texas
    • Dallas, Texas, United States, 75231
      • Research Institute Of Dallas
  • Washington
    • Tacoma, Washington, United States, 98405
      • MultiCare Institute for Research & Innovation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: RAD011 40 milligrams per kilogram (mg/kg); Participants were administered 40 mg/kg of RAD011 orally daily with food.	Drug: RAD011; Cannabidiol Oral Solution (containing synthetic cannabidiol)
Active Comparator: RAD011 20 mg/kg; Participants were administered 20 mg/kg of RAD011 orally daily with food.	Drug: RAD011; Cannabidiol Oral Solution (containing synthetic cannabidiol)
Active Comparator: RAD011 10 mg/kg; Participants were administered 10 mg/kg of RAD011 orally daily with food.	Drug: RAD011; Cannabidiol Oral Solution (containing synthetic cannabidiol)
Placebo Comparator: Placebo; Participants were administered a placebo matching to RAD011, orally daily with food.	Drug: Placebo; Matching Placebo for RAD011

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline, Week 34

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline, Week 34
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.	Baseline, Week 34
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.	Baseline, Week 34

Collaborators and Investigators

• Sponsor: Radius Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): April 13, 2022
• Primary Completion (Actual): October 6, 2022
• Study Completion (Actual): October 31, 2022

Study Registration Dates

• First Submitted: September 22, 2021
• First Submitted That Met QC Criteria: October 18, 2021
• First Posted (Actual): October 28, 2021

Study Record Updates

• Last Update Posted (Actual): October 19, 2023
• Last Update Submitted That Met QC Criteria: September 26, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: SCOUT-015

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No