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

NCT06420297 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 2 other identifiers: ACP-101-3032023-506201-19-00
• Study Type: interventional
• Target: 160
• Locations: 6 countries
• Sites: 30

Brief Summary

To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS

Conditions

Hyperphagia in Prader-Willi Syndrome

Keywords

Opel-label extension; safety; tolerability

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions

  Treatment-emergent adverse events (TEAEs) and device incidents or device malfunctions, TEAEs leading to discontinuation, TEAEs related to study drug, TEAEs by maximum severity, fatal TEAEs, treatment-emergent SAEs, and treatment-emergent SAEs related to study drug will all be summarized for all subjects as well as by previous treatment group.

  Baseline to 36 months

Study Arms (1)

Drug: Carbetocin

EXPERIMENTAL

Carbetocin nasal spray 3.2 mg three times daily (TID)

Drug: Carbetocin

Interventions

Carbetocin DRUG

Carbetocin nasal spray 3.2 mg three times daily (TID)

Also known as: ACP-101

Drug: Carbetocin

Eligibility Criteria

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

You may qualify if:

• Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302
• Met all entry criteria for the antecedent study
• May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator.
• 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:

• Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
• History of suicide attempt or inpatient psychiatric hospitalization
• Has a clinically significant abnormality in vital signs at Baseline
• Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
• Has developed a clinically significant ECG finding during the antecedent study
• Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study

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 Kansas City

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 - Hopital des Enfants

Toulouse, Cedex 9 Occitanie, 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, 28007, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29009, Spain

Location

University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital

Birmingham, B9 5SS, United Kingdom

Location

Royal Hospital for Children Glasgow Clinical

Glasgow, G51 4TF, United Kingdom

Location

Barts Health NHS Trust - The Royal London Hospital

London, E1 1B6, United Kingdom

Location

MeSH Terms

Interventions

carbetocin

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 7, 2024
• First Posted: May 20, 2024
• Study Start: March 11, 2024
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): June 1, 2029
• Last Updated: December 3, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

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