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

NCT06173531 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: ACP-101-3022023-506200-24-00
• Study Type: interventional
• Target: 170
• Locations: 6 countries
• Sites: 30

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)

Conditions

Hyperphagia in Prader-Willi Syndrome

Keywords

Randomized; Placebo-controlled

Outcome Measures

Primary Outcomes (1)

• Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score

  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.

  Baseline to Week 12

Secondary Outcomes (2)

• Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS

  Baseline to Week 12

• Clinical Global Impression-Change (CGI-C) for PWS score at Week 12

  Score at Week 12

Study Arms (2)

Carbetocin

EXPERIMENTAL

Carbetocin nasal spray 3.2 mg three times daily (TID)

Drug: Carbetocin

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Carbetocin DRUG

Carbetocin nasal spray 3.2 mg three times daily (TID)

Also known as: ACP-101

Carbetocin

Placebo DRUG

Placebo given TID, identical in appearance respective to carbetocin treatment

Placebo

Eligibility Criteria

• Age: 5 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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
• 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)

Sponsors & Collaborators

• ACADIA Pharmaceuticals Inc.: lead

Study Sites (30)

Children's of Alabama

Birmingham, Alabama, 35233, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85006, United States

Location

University of California Irvine

Orange, California, 92697, United States

Location

Stanford University School of Medicine

Palo Alto, California, 94304, United States

Location

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

SSM Health/Saint Louis University

St Louis, Missouri, 63104, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11219, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

UPMC-Children's Hospital Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Vanderbilt Clinical Research Center

Nashville, Tennessee, 37232, United States

Location

Cook Children's Health Care System

Fort Worth, Texas, 79104, United States

Location

Christus Children's

San Antonio, Texas, 78207, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Alberta Diabetes Institute

Edmonton, Alberta, T6G 2E1, Canada

Location

CHU Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants

Toulouse, 31059, France

Location

KJF Klinik Josefinum gGmbH

Augsburg, 86154, Germany

Location

Universitätsklinikum Essen

Essen, 45147, Germany

Location

Parc Taulí Hospital Universitari

Barcelona, 08208, Spain

Location

Hospital General Universitario Gregorio Marañón

Madrid, 28009, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29010, Spain

Location

University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital

Birmingham, B9 5SS, United Kingdom

Location

Royal Hospital for Children Glasgow Clinical Research Facility

Glasgow, G51 4TF, United Kingdom

Location

Barts Health NHS Trust - The Royal London Hospital

London, E1 1BB, United Kingdom

Location

MeSH Terms

Interventions

carbetocin

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2023
• First Posted: December 15, 2023
• Study Start: November 27, 2023
• Primary Completion: October 1, 2025
• Study Completion: November 1, 2025
• Last Updated: September 10, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); DE (2); CA (2); ES (3); GB (3); US (19)