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

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• 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 [Срок оценки: Baseline to 36 months]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 19 центров

• Children's of Alabama — Birmingham
• Phoenix Children's Hospital — Phoenix
• University of California Irvine — Orange
• Stanford University School of Medicine — Palo Alto
• Rady Children's Hospital San Diego — San Diego
• Children's Hospital Colorado — Aurora
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
• University of Iowa — Iowa City
• … и ещё 11 центров

Испания · 3 центра

• Parc Taulí Hospital Universitari — Barcelona
• Hospital General Universitario Gregorio Marañón — Madrid
• Hospital Regional Universitario de Málaga — Málaga

Великобритания · 3 центра

• University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital — Birmingham
• Royal Hospital for Children Glasgow Clinical — Glasgow
• Barts Health NHS Trust - The Royal London Hospital — London

Канада · 2 центра

• Alberta Diabetes Institute — Edmonton
• CHU Sainte-Justine — Montreal

Германия · 2 центра

• KJF Klinik Josefinum gGmbH — Augsburg
• Universitätsklinikum Essen — Essen

Франция · 1 центр

• Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital des Enfants — Toulouse

Идентификаторы

NCT: NCT06420297 · ACP-101-303 · 2023-506201-19-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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