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

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Hyperphagia in Prader-Willi Syndrome

In brief

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

Key facts

Study ID

NCT06173531

Run by

ACADIA Pharmaceuticals Inc.

People needed

170

Starts

2023-11-27

Expected to finish

2025-11-01

Last updated by the study team

2025-09-10

Who can join

Age: 5 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

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
• 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.

Where it is running

• 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
• Parc Taulí Hospital Universitari — Barcelona, Spain

Full record on ClinicalTrials.gov

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