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NCT06173531·Phase 3·INTERVENTIONAL

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

Status

Active, not recruiting

Phase

Phase 3

Enrollment

170

Locations

30

Results

Not posted

Publications

1

Study summary

What the protocol is testing.

12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)

Full 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.

Interventions

Treatment arms and agents.

DRUG

Carbetocin

Carbetocin nasal spray 3.2 mg three times daily (TID)

DRUG

Placebo

Placebo given TID, identical in appearance respective to carbetocin treatment

Timeline

From registration to results.

  1. First posted

    Dec 15, 2023

  2. Study start

    Nov 27, 2023

  3. Primary completion

    Oct 2025

  4. Study completion

    Nov 2025

  5. Results posted

    Not reported

  6. Registry updated

    Sep 10, 2025

Outcomes

What the study measures.

Primary 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.

Secondary outcomes

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

Who can take part.

Minimum age
5 Years
Maximum age
30 Years
Sex
ALL
Healthy volunteers
No

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

30 registered sites.

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

Related trials

More studies on Carbetocin.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.