OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
- Registration Number
- NCT06420297
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Brief Summary
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS
- 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 160
- 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.
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Drug: Carbetocin Carbetocin Carbetocin nasal spray 3.2 mg three times daily (TID)
- Primary Outcome Measures
Name Time Method 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 Baseline to 36 months 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.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (25)
KJF Klinik Josefinum gGmbH
🇩🇪Augsburg, Germany
Children's of Alabama
🇺🇸Birmingham, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
University of California Irvine
🇺🇸Orange, California, United States
Stanford University School of Medicine
🇺🇸Palo Alto, California, United States
Rady Children's Hospital San Diego
🇺🇸San Diego, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Mercy Kansas City
🇺🇸Kansas City, Missouri, United States
SSM Health/Saint Louis University
🇺🇸Saint Louis, Missouri, United States
Maimonides Medical Center
🇺🇸Brooklyn, New York, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
UPMC-Children's Hospital Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Vanderbilt Clinical Research Center
🇺🇸Nashville, Tennessee, United States
Cook Children's Health Care System
🇺🇸Fort Worth, Texas, United States
Christus Children's
🇺🇸San Antonio, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Alberta Diabetes Institute
🇨🇦Edmonton, Alberta, Canada
CHU Sainte-Justine
🇨🇦Montreal, Quebec, Canada
University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
🇬🇧Birmingham, United Kingdom
Royal Hospital for Children Glasgow Clinical
🇬🇧Glasgow, United Kingdom
Barts Health NHS Trust - The Royal London Hospital
🇬🇧London, United Kingdom