A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Status: Active_not_recruiting
Location: See all (30) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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)
Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 30
Healthy Volunteers: f
View:
• Male or female and 5 through 30 years of age
• Prader-Willi syndrome with a documented disease-causing mutation
• Increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)
• HQ-CT total score of ≥13 at Screening and Baseline
• CGI-S score for hyperphagia in PWS of ≥4 at Screening and Baseline
• Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits
Locations
United States
Alabama
Children's of Alabama
Birmingham
Arizona
Phoenix Children's Hospital
Phoenix
California
University of California Irvine
Orange
Stanford University School of Medicine
Palo Alto
Rady Children's Hospital San Diego
San Diego
Colorado
Children's Hospital Colorado
Aurora
Iowa
University of Iowa
Iowa City
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Massachusetts
Boston Children's Hospital
Boston
Missouri
Children's Mercy Hospital
Kansas City
SSM Health/Saint Louis University
St Louis
New York
Maimonides Medical Center
Brooklyn
Ohio
Nationwide Children's Hospital
Columbus
Pennsylvania
UPMC-Children's Hospital Pittsburgh
Pittsburgh
Tennessee
Vanderbilt Clinical Research Center
Nashville
Texas
Cook Children's Health Care System
Fort Worth
Christus Children's
San Antonio
Utah
University of Utah
Salt Lake City
Washington
Seattle Children's Hospital
Seattle
Other Locations
Canada
Alberta Diabetes Institute
Edmonton
CHU Sainte Justine
Montreal
France
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse
Germany
KJF Klinik Josefinum gGmbH
Augsburg
Universitätsklinikum Essen
Essen
Spain
Parc Taulí Hospital Universitari
Barcelona
Hospital General Universitario Gregorio Marañón
Madrid
Hospital Regional Universitario de Málaga
Málaga
United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
Birmingham
Royal Hospital for Children Glasgow Clinical Research Facility
Glasgow
Barts Health NHS Trust - The Royal London Hospital
London
Time Frame
Start Date: 2023-11-27
Completion Date: 2025-11
Participants
Target number of participants: 170
Treatments
Experimental: Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)
Placebo_comparator: Placebo
Placebo
Related Therapeutic Areas
Sponsors
Leads: ACADIA Pharmaceuticals Inc.