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

NCT05098509 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: SCOUT-015
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 8

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.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)

  Baseline, Week 34

• Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)

  Baseline, Week 34

• Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)

  Baseline, Week 34

Study Arms (4)

RAD011 40 milligrams per kilogram (mg/kg)

ACTIVE COMPARATOR

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

Drug: RAD011

RAD011 20 mg/kg

ACTIVE COMPARATOR

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

Drug: RAD011

RAD011 10 mg/kg

ACTIVE COMPARATOR

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

Drug: RAD011

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

RAD011 DRUG

Cannabidiol Oral Solution (containing synthetic cannabidiol)

RAD011 10 mg/kg; RAD011 20 mg/kg; RAD011 40 milligrams per kilogram (mg/kg)

Placebo DRUG

Matching Placebo for RAD011

Placebo

Eligibility Criteria

• Age: 8 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Radius Pharmaceuticals, Inc.: lead

Study Sites (8)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Rady Children's Hospital

San Diego, California, 92123, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11219, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Research Institute of Dallas

Dallas, Texas, 75231, United States

Location

MultiCare Institute for Research & Innovation

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Limitations and Caveats

The study has been voluntarily terminated by the sponsor due to change in corporate priorities, for reasons other than safety. Due to early termination of study, no efficacy analyses were done.

Results Point of Contact

• Title: Ellie Ratigan
• Organization: Radius Pharmaceuticals, Inc.

scout015@radiuspharm.com

617-444-1800

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2021
• First Posted: October 28, 2021
• Study Start: April 13, 2022
• Primary Completion: October 6, 2022
• Study Completion: October 31, 2022
• Last Updated: October 19, 2023
• Results First Posted: October 19, 2023

Record last verified: 2023-09

Locations

US (8)