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

NCT ID: NCT06173531

Last Updated: 2025-09-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

170 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-27

Study Completion Date

2025-11-30

Brief Summary

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

Detailed Description

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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.

Conditions

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Hyperphagia in Prader-Willi Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Carbetocin

Carbetocin nasal spray 3.2 mg three times daily (TID)

Group Type EXPERIMENTAL

Carbetocin

Intervention Type DRUG

Carbetocin nasal spray 3.2 mg three times daily (TID)

Placebo

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo given TID, identical in appearance respective to carbetocin treatment

Interventions

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Carbetocin

Carbetocin nasal spray 3.2 mg three times daily (TID)

Intervention Type DRUG

Placebo

Placebo given TID, identical in appearance respective to carbetocin treatment

Intervention Type DRUG

Other Intervention Names

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ACP-101

Eligibility Criteria

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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)
Minimum Eligible Age

5 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ACADIA Pharmaceuticals Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's of Alabama

Birmingham, Alabama, United States

Site Status

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

University of California Irvine

Orange, California, United States

Site Status

Stanford University School of Medicine

Palo Alto, California, United States

Site Status

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

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

Chicago, Illinois, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

SSM Health/Saint Louis University

St Louis, Missouri, United States

Site Status

Maimonides Medical Center

Brooklyn, New York, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

UPMC-Children's Hospital Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt Clinical Research Center

Nashville, Tennessee, United States

Site Status

Cook Children's Health Care System

Fort Worth, Texas, United States

Site Status

Christus Children's

San Antonio, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Alberta Diabetes Institute

Edmonton, Alberta, Canada

Site Status

CHU Sainte Justine

Montreal, Quebec, Canada

Site Status

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

Toulouse, , France

Site Status

KJF Klinik Josefinum gGmbH

Augsburg, , Germany

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Parc Taulí Hospital Universitari

Barcelona, , Spain

Site Status

Hospital General Universitario Gregorio Marañón

Madrid, , Spain

Site Status

Hospital Regional Universitario de Málaga

Málaga, , Spain

Site Status

University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital

Birmingham, , United Kingdom

Site Status

Royal Hospital for Children Glasgow Clinical Research Facility

Glasgow, , United Kingdom

Site Status

Barts Health NHS Trust - The Royal London Hospital

London, , United Kingdom

Site Status

Countries

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United States Canada France Germany Spain United Kingdom

Other Identifiers

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2023-506200-24-00

Identifier Type: CTIS

Identifier Source: secondary_id

ACP-101-302

Identifier Type: -

Identifier Source: org_study_id

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