Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome.
IRAS ID
1008386
Contact name
Heather Bradley
Contact email
Sponsor organisation
Acadia Pharmaceuticals Inc.
Research summary
Prader-Willi Syndrome (PWS) is a genetic disorder that can cause problems with physical, mental, and behavioural development. People with PWS often have a symptom of extreme hunger and wanting to eat but never feeling full. This is called hyperphagia. This trial is being done to learn how a trial drug called carbetocin works in reducing uncontrollable hunger in people with PWS.
At the time of this trial, doctors may recommend treatments to manage symptoms of PWS, but there are no approved medicines for hyperphagia in people with PWS. Previous studies of carbetocin as a spray into the nose (also called nasal spray) showed less hyperphagia and anxiousness in some participants with PWS who took carbetocin.
Participants will be randomly assigned by a computer program to one of 2 groups:
Group 1: carbetocin 3.2 mg three times a day as a nasal spray for up to 12 weeks.
Group 2: placebo three times a day as a nasal spray for up to 12 weeks.
A placebo looks like the trial drug but does not have medicine in it. During the trial no one will know which treatment participants will be taking.
170 participants diagnosed with PWS between the ages of 5 to 30 years of age will take part at approximately 36 centres in several countries.
The trial will have three distinct periods:
- a screening period, involving 1 or more visits to the hospital during a 3-week period to check if the participant can join the trial.
- a trial treatment period of 12 weeks, involving 3 visits to the hospital and a remote visit.
- a follow-up telephone call 4 weeks after the participant finishes or stops taking the trial treatment.The main goal of this trial is to find out how effective is treatment with carbetocin nasal spray in comparison to placebo on hyperphagia in participants with PWS.
Trial duration for an individual participant is approximately 19 weeks.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
23/YH/0272
Date of REC Opinion
30 Jan 2024
REC opinion
Further Information Favourable Opinion