DRUG
Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)
Status
Active, not recruiting
Phase
Phase 3
Enrollment
170
Locations
30
Results
Not posted
Publications
1
Study 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)
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.
Interventions
DRUG
Carbetocin nasal spray 3.2 mg three times daily (TID)
DRUG
Placebo given TID, identical in appearance respective to carbetocin treatment
Timeline
First posted
Dec 15, 2023
Study start
Nov 27, 2023
Primary completion
Oct 2025
Study completion
Nov 2025
Results posted
Not reported
Registry updated
Sep 10, 2025
Outcomes
Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
Time frame · 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.
Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS
Time frame · 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
Time frame · 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).
Eligibility
Inclusion Criteria * 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 Exclusion Criteria * Genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS * An active upper respiratory infection at the Screening visit or the Baseline visit * Any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma * History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary * Nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study. * Unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study * Clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit * Used prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary. * Started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening. * Used oxytocin, desmopressin (DDAVP), tesofensine, diazoxide choline, melanocortin-4 receptor (MC4R) agonists (e.g., setmelanotide), or any medication approved to treat hyperphagia within 6 months prior to the Baseline visit * Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder * History of suicide attempt or inpatient psychiatric hospitalization * New food-related interventions, including environment or dietary restrictions, within 1 month prior to the Screening visit or during the Screening period (i.e., before the Baseline visit) Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.
Study locations
Canada · France · Germany · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
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
SSM Health/Saint Louis University
St Louis, Missouri, United States
Maimonides Medical Center
Brooklyn, New York, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
UPMC-Children's Hospital Pittsburgh
Pittsburgh, Pennsylvania, United States
Vanderbilt Clinical Research Center
Nashville, Tennessee, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
Christus Children's
San Antonio, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Alberta Diabetes Institute
Edmonton, Alberta, Canada
CHU Sainte Justine
Montreal, Quebec, Canada
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, France
KJF Klinik Josefinum gGmbH
Augsburg, Germany
Universitätsklinikum Essen
Essen, Germany
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