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

NCT ID: NCT06420297

Last Updated: 2025-12-03

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

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-11

Study Completion Date

2029-06-30

Brief Summary

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

Detailed Description

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This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.

Conditions

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Hyperphagia in Prader-Willi Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Drug: 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)

Interventions

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Carbetocin

Carbetocin nasal spray 3.2 mg three times daily (TID)

Intervention Type DRUG

Other Intervention Names

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ACP-101

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
* 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
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 Kansas City

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 - Hopital des Enfants

Toulouse, Cedex 9 Occitanie, 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

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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2023-506201-19-00

Identifier Type: CTIS

Identifier Source: secondary_id

ACP-101-303

Identifier Type: -

Identifier Source: org_study_id

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