Long-Term Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

  • Research type

    Research Study

  • Full title

    A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

  • IRAS ID

    1009499

  • Contact name

    Heather Bradley

  • Contact email

    hbradley@acadia-pharm.com

  • Sponsor organisation

    Acadia Pharmaceuticals Inc.

  • Clinicaltrials.gov Identifier

    NCT06420297

  • 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.
    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.

    The purpose of this trial is to investigate the long-term safety and tolerability of carbetocin nasal spray for a symptom of PWS called hyperphagia. This long-term study will include up to 160 participants who completed the Week 12 visit of the previous ACP-101-302 study.

    In this long-term study all participants will receive the study drug carbetocin 3.2 mg three times a day as a nasal spray for up to 156 weeks.

    The trial will have 2 distinct periods:

    - a trial treatment period of 156 weeks, involving 10 visits to the hospital and 5 remote visits.
    - a follow-up telephone call 4 weeks after the participant finishes or stops taking the trial treatment.

    Trial duration for an individual participant is approximately 160 weeks or until the Sponsor Acadia Pharmaceuticals Inc., terminates the study.

  • REC name

    Yorkshire & The Humber - Leeds East Research Ethics Committee

  • REC reference

    24/YH/0081

  • Date of REC Opinion

    23 May 2024

  • REC opinion

    Further Information Favourable Opinion