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

A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

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

Study Overview

• Status: Enrolling by invitation
• Conditions: Hyperphagia in Prader-Willi Syndrome
• Intervention / Treatment: Drug: Carbetocin

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 160
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • New York
    • Brooklyn, New York, United States, 11219
      • Maimonides Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt Clinical Research Center
  • Texas
    • Fort Worth, Texas, United States, 79104
      • Cook Children's Health Care System

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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

Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Drug: Carbetocin; Carbetocin nasal spray 3.2 mg three times daily (TID)	Drug: Carbetocin; Carbetocin nasal spray 3.2 mg three times daily (TID); Other Names: ACP-101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: ACADIA Pharmaceuticals Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 11, 2024
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): June 1, 2029

Study Registration Dates

• First Submitted: May 7, 2024
• First Submitted That Met QC Criteria: May 14, 2024
• First Posted (Actual): May 20, 2024

Study Record Updates

• Last Update Posted (Actual): June 4, 2024
• Last Update Submitted That Met QC Criteria: June 3, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Keywords: tolerability; safety; Opel-label extension
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Signs and Symptoms, Digestive; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Imprinting Disorders; Syndrome; Prader-Willi Syndrome; Hyperphagia; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Carbetocin
• Other Study ID Numbers: ACP-101-303; 2023-506201-19-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No