Treatment of Hyperphagia in Patients with Prader-Willi Syndrome

Summary:  The study will consist of two parts:

Part 1: Two dose levels of XXXX or matching placebo will be administered.

Patients will be randomized in a 1:1:1 ratio to one of the following treatment arms.

Treatment Arm A: XXXX 200 mg administered twice a day (BID) for 12 weeks (n=12)

Treatment Arm B: XXXX 800 mg administered BID for 12 weeks (n=12)

Treatment Arm C: Matching placebo administered BID for 12 weeks (n=12)

Randomization will be stratified by:

• TBD

Following an independent Data and Safety Monitoring Committee (DSMC) review, the study will continue with one active dose level per DSMC recommendation.


Part 2: One dose level of XXXX or matching placebo will be administered.

Patients will be randomized in a 1:1 ratio to one of the following treatment arms.

Treatment Arm A: DSMC recommended XXXX dose administered BID for 12 weeks (n=57)

Treatment Arm B: Matching placebo administered BID for 12 weeks (n=57)

Type of study (PIC, Recruiting):  Recruiting

Commercial Study - Site Identification form required to be completed, please find Site ID form uploaded to study documents link below. 

Recruitment Duration: 16/12/2024 - 15/08/2025

Practice Activity: See part B and Schedule of Events 

Practice Eligibility Criteria: GP practices within East Midlands

Practice Target: None at this time

Is this Study Applicable for RSI Scheme?  

Yes, applicable for RSI scheme

Research Costs: 

Commercial research studies follow a national tariff guideline. Click here for more information on the tariff or download the tariff by clicking here