Clinical trial NCT05098509: 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
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Prader-Willi Syndrome
Lead sponsor: Radius Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-10-28
Enrollment: 4
Sex: ALL
Minimum age (years): 8
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change From Baseline to Week 34 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Questionnaire
timeFrame: Baseline, Week 34
Secondary outcomes:
measure: Change From Baseline to Week 34 in Prader-Willi Syndrome (PWS)- Associated Irritability Using the Aberrant Behavior Checklist (ABC) Questionnaire - Irritability Subscale (ABC-I)
timeFrame: Baseline, Week 34
measure: Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Change (CGI-C)
timeFrame: Baseline, Week 34
measure: Change From Baseline to Week 34 in Hyperphagia as Measured by the Clinician Global Impression of Severity (CGI-S)
timeFrame: Baseline, Week 34
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Brooklyn
country: United States
facility: Maimonides Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Research Institute of Dallas
state: Texas
city: Tacoma
country: United States
facility: MultiCare Institute for Research & Innovation
state: Washington
Study officials:
References: