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

Radius Health

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Prader-Willi Syndrome

Treatments: Drug: Placebo; Drug: RAD011

Study type: Interventional

Funder types: Industry

Identifiers

NCT05098509

SCOUT-015

Details and patient eligibility

About

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.

Enrollment: 4 patients

Sex: All

Ages: 8 to 65 years old

Volunteers: No 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, 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

4 participants in 4 patient groups, including a placebo group

RAD011 40 milligrams per kilogram (mg/kg)

Active Comparator group

Description:

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

Treatment:

Drug: RAD011

RAD011 20 mg/kg

Active Comparator group

Description:

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

Treatment:

Drug: RAD011

RAD011 10 mg/kg

Active Comparator group

Description:

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

Treatment:

Drug: RAD011

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo