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

Phase 3TerminatedNCT05098509

Radius Pharmaceuticals, Inc.4 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Prader-Willi Syndrome

Interventions

RAD011DRUG

Cannabidiol Oral Solution (containing synthetic cannabidiol)

PlaceboDRUG

Matching Placebo for RAD011

Eligibility

Sex: ALLMin age: 8 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (8)

Children's Hospital Los Angeles

Los Angeles, California, United States

Rady Children's Hospital

San Diego, California, United States

University of Iowa

Iowa City, Iowa, United States

Maimonides Medical Center

Brooklyn, New York, United States

Outcomes

Primary Outcomes

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.

Time frame: Baseline, Week 34

Secondary Outcomes

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.

Time frame: 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.

Time frame: 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.

Data from ClinicalTrials.gov

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