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

Hyperphagia in Prader-Willi Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06420297
• Other study ID #: ACP-101-303
• Secondary ID: 2023-506201-19-0
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: March 11, 2024
• Est. completion date: June 2029

Study information

• Verified date: June 2024
• Source: ACADIA Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 160
• Est. completion date: June 2029
• Est. primary completion date: May 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 30 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hyperphagia
• Hyperphagia in Prader-Willi Syndrome
• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)

Locations

Country	Name	City	State
United States	Maimonides Medical Center	Brooklyn	New York
United States	Cook Children's Health Care System	Fort Worth	Texas
United States	University of Iowa	Iowa City	Iowa
United States	Vanderbilt Clinical Research Center	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
ACADIA Pharmaceuticals Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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