Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Interventions
- Drug: Placebo
- Registration Number
- NCT06173531
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Brief Summary
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
- Detailed Description
This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 170
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Carbetocin Carbetocin Carbetocin nasal spray 3.2 mg three times daily (TID) Placebo Placebo Placebo
- Primary Outcome Measures
Name Time Method Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score Baseline to Week 12 The HQ-CT is a nine-item questionnaire designed to be completed by caregivers of subjects with PWS. It is a revision of the 11-item HPWSQ-R and has been further validated. The Foundation for Prader-Willi Research has made the HQ-CT available for clinical studies in PWS, and it is the consensus instrument within the PWS research community for measuring observable behaviors that stem from subjects' excessive drive to eat.
The HQ-CT should be completed by the same caregiver throughout the study. The HQ-CT will be administered to the caregiver by a rater using standardized prompts. The Food Safe Zone should be administered immediately before administration of the HQ-CT.
A higher score on the HQ-CT indicates greater severity of hyperphagia.
- Secondary Outcome Measures
Name Time Method Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS Baseline to Week 12 The CGI-S is a rating scale that records a clinician's global impression of the current severity of illness on a seven-point scale, using a range of responses from 1 (normal) to 7 (among the most severely ill subjects).
Clinical Global Impression-Change (CGI-C) for PWS score at Week 12 Score at Week 12 The CGI-C is a rating scale that records a clinician's global impression of change in severity of illness, using a range of responses from 1 (very much improved) to 7 (very much worse).
Trial Locations
- Locations (30)
Children's of Alabama
🇺🇸Birmingham, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
University of California Irvine
🇺🇸Orange, California, United States
Stanford University School of Medicine
🇺🇸Palo Alto, California, United States
Rady Children's Hospital San Diego
🇺🇸San Diego, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Scroll for more (20 remaining)Children's of Alabama🇺🇸Birmingham, Alabama, United States