A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study of RAD011 in Prader-Willi Syndrome
Research type
Research Study
Full 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
IRAS ID
1004889
Contact name
Akbar Khan
Contact email
Sponsor organisation
Radius Pharmaceuticals, Inc.
Eudract number
2021-005331-23
Clinicaltrials.gov Identifier
Research summary
Research Summary
Prader-Willi Syndrome (PWS) is a multifaceted neuro developmental disorder and the most common genetic syndrome associated with obesity. Clinically, PWS patients suffer a complex pattern of physical, behavioural, endocrine and intellectual deficiencies. However, it is the appetite behaviour classified as hyperphagia (persistent uncontrolled feeling of hunger leading to excessive food consumption) in PWS that is the most life threatening and one of its greatest unmet medical needs. The investigational drug RAD011 (synthetic, pharmaceutical grade cannabidiol oral solution) is being studied as a treatment for hyperphagia as well as for PWS-related irritability and overall behaviours. The main purpose of this study is to determine if RAD011 is safe, tolerated and if it can decrease hyperphagia in PWS patients, as well as to assess its effect on PWS-related irritability and overall problem behaviours.
The study is divided in two parts (phase 2 and phase 3) each one consisting of six similar periods- Screening, Tolerability, Dose Escalation, Maintenance, Taper and Follow-up. Each phase will last approximately 40 weeks and will include a total of 16 study visits. Three RAD011 doses will be tested during phase 2. The doses with the best safety and tolerability results, as determined by a safety monitoring committee, will be further tested during phase 3. Each patient will be enrolled in only one of the study phases and will be assigned by chance to receive either one of the RAD011 doses or the placebo (inactive investigational drug). Approximately 220 patients worldwide will participatey.
Currently, there is not an approved therapy that effectively targets the hyperphagia in these patients. Previous clinical findings across other clinical indications, as well as existing safety and tolerability data, support the use of RAD011 to address hyperphagia in PWS patients in an attempt to alter the subsequent severe obesity and related complications associated with this syndrome.Summary of Results
This study was voluntarily terminated by the Sponsor for reasons other than safety prior to achieving the planned sample size; only 13 patients were screened, 7 patients were enrolled, and 4 patients were randomised in the Phase 2 part of the study in the United States of America (USA). No patients were enrolled or randomised elsewhere in the world.
Subject enrollment was ceased, and Early Termination (ET)/End of Study (EOS) visits were scheduled for all randomised subjects. Patients who were participating in the Tolerability Period but not yet randomised were screen failed. As a result, the planned analyses described in the protocol did not occur.REC name
South Central - Oxford A Research Ethics Committee
REC reference
22/SC/0140
Date of REC Opinion
22 Sep 2022
REC opinion
Further Information Favourable Opinion