A Study of Pitolisant in Patients With Prader-Willi Syndrome
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: Pitolisant tablet, Placebo tablet.
- Кому может быть актуально
- Состояния в реестре: Prader-Willi Syndrome. Базовые параметры: от 6 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США, Австралия, Бельгия, Канада, Дания +8
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome
Обзор
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome. Secondary objectives include assessing the impact of pitolisant on: Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
Подробное описание
The study will consist of an up to 45-day Screening/Baseline Period, a Double-Blind Treatment Period, and an optional Open-Label Extension Period.
After completion of all Baseline assessments, patients who meet all eligibility criteria will be randomized 1:1 to receive once daily pitolisant or matching placebo. During the Double-Blind Treatment Period, in-person visits will be at Day 29, Day 57, and Day 77. Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug.
During the optional Open-Label Extension Period, in-person visits will be at Day 113, Day 260, and Day 441. Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant.
Вмешательства
- Препарат Pitolisant tablet
Pitolisant tablet - Другое Placebo tablet
Placebo tablet
Первичные конечные точки
- Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
Вторичные конечные точки (8)
- Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS) [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS) [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in overall severity of irritable and disruptive behaviors as measured by the Caregiver Global Impression of Severity (CaGI-S) for Irritable and/or Disruptive Behaviors [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in severity of hyperphagia as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), in conjunction with the Food Safe Zone Questionnaire (FSZQ) [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in severity of EDS as measured by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD [parent/caregiver version]) total score [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Change in severity of other behavioral problems as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, and Stereotypic Behavior Domains [Срок оценки: Baseline and end of the Double Blind Treatment Period (Day 77)]
- Percentage of patients reporting TEAEs [Срок оценки: Baseline up to Day 441]
Критерии участия
Критерии включения
- Genetically confirmed diagnosis of PWS
- Excessive daytime sleepiness
- Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
- In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
Критерии исключения
- Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled
- Has a diagnosis of hypersomnia due to another sleep/medical disorder
- Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Рандомизированное
- Модель
- Параллельные группы
- Маскирование
- Четверное слепое
- Основная цель
- Лечение
Центры проведения
США · 21 центр
- Santa Monica Clinical Trials — Los Angeles
- Center of Excellence in Diabetes and Endocrinology — Sacramento
- Rady Children's Hospital - Scan Diego — San Diego
- Colorado Children's Hospital — Aurora
- Nemours Children's Hospital — Wilmington
- Atlanta Diabetes Associates — Atlanta
- Emory University School of Medicine — Atlanta
- Rare Disease Research — Atlanta
- … и ещё 13 центров
Италия · 6 центров
- Azienda Ospedaliero Universitaria A Meyer — Florence
- Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN — Genova
- Ospedale San Raffaele S.r.l. - PPDS — Milan
- Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIP — Padova
- Ospedale Pediatrico Bambino Gesù IRCCS — Roma
- IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN — Trieste
Австралия · 4 центра
- Queensland Children's Hospital — Brisbane
- Royal Prince Alfred Hospital — Camperdown
- Sydney Children's Hospital — Randwick
- Children's Hospital at Westmead — Westmead
Румыния · 4 центра
- Institutul National de Endocrinologie C. I. Parhon — Bucharest
- Institutul National de Endocrinologie C. I. Parhon — Bucharest
- National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu" — Bucharest
- Louis Turcanu Emergency Clinical Hospital for Children — Timișoara
Испания · 4 центра
- Corporacio Sanitaria Parc Tauli, Sabadell — Barcelona
- Hospital Sant Joan de Deu — Barcelona
- Hospital Universitario 12 de Octubre — Madrid
- Universitario Virgen de la Victoria — Málaga
Великобритания · 4 центра
- Fulbourn Hospital — Cambridge
- Hull Royal Infirmary — Hull
- Ninewells Hospital - PPDS — Dundee
- Royal Hospital for Children and Young People — Edinburgh
Канада · 2 центра
- AMNDX Inc. — Thornhill
- Jodha Tishon Inc. — Toronto
Дания · 2 центра
- Aarhus University Hospital — Aarhus
- Rigshospitalet — Copenhagen
Франция · 2 центра
- CHU d'Angers — Angers
- CHU de Toulouse-Hôpital Des Enfants — Toulouse
Польша · 2 центра
- Samodzielny Publiczny Szpital Kliniczny — Szczecin
- Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego — Wroclaw
Бельгия · 1 центр
- UZ Brussels — Jette
Германия · 1 центр
- University Hospital Essen — Essen
Швеция · 1 центр
- Karolinska Universitetssjukhuset Solna — Solna
Идентификаторы
NCT: NCT06366464 · HBS-101-CL-312