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

Launched by ACADIA PHARMACEUTICALS INC. · May 14, 2024

Keywords

ClinConnect Summary

This study is a long-term, open-label extension to test the safety and tolerance of carbetocin nasal spray (3.2 mg three times a day) in people with Prader-Willi syndrome who have hyperphagia (unwanted hunger). It follows an earlier trial and all participants in this extension know they are receiving the drug. The goal is to monitor safety over up to about three years, including any adverse events, serious problems, or device issues, in a single-group design (no placebo).

Who can participate? If you are between ages 5 and 30, have completed the previous ACP-101-302 study, and caregivers are willing to participate and follow the study visits, you may be eligible. Exclusions include a history of certain brain or heart conditions, epilepsy or psychosis, a suicide history, and certain abnormal heart rhythm findings. The study is happening at many centers in the United States, Canada, Europe, and other countries, and is sponsored by ACADIA Pharmaceuticals. Important points for families: you’ll need to commit to regular visits, the treatment is a nasal spray taken three times daily, and the researchers will closely watch for any adverse events or device problems. The trial is currently enrolling by invitation, with no results available yet, and is expected to continue through 2029.

Gender

ALL

Eligibility criteria

• 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.