A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
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: AOC-1020, Placebo.
- Кому может быть актуально
- Состояния в реестре: Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1. Базовые параметры: 16 лет — 70 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, Канада, Дания, Франция, Германия +5
- 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 Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
Обзор
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
Подробное описание
The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).
Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.
After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety.
An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Вмешательства
- Препарат AOC-1020
Del-brax will be administered by intravenous (IV) infusion. - Препарат Placebo
Placebo will be administered by intravenous (IV) infusion.
Первичные конечные точки
- Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN) [Срок оценки: Baseline through Week 78]
Вторичные конечные точки (10)
- Change from Baseline to Week 78 in 10MWRT [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Timed Up-and-Go (TUG) [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in PROMIS Fatigue [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS) [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C) [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L [Срок оценки: Baseline through Week 78]
- Change from Baseline to Week 78 in Serum CK [Срок оценки: Baseline through Week 78]
Критерии участия
Критерии включения
- Clinical and genetic diagnosis of FSHD1 or FSHD2
- Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
- Adequate muscle strength based on QMT composite score
Критерии исключения
- Breastfeeding, pregnancy, or intent to become pregnant during the study
- Unwilling or unable to comply with contraceptive requirements
- Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- Blood Pressure > 140/90 mmHg at Screening
- Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
- Treatment with an oligonucleotide within 9 months of 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 · 14 центров
- University of California Irvine — Orange
- Stanford University — Palo Alto
- University of Colorado — Denver
- University of Florida — Gainesville
- University of Iowa — Iowa City
- Kansas University Medical Center — Kansas City
- Kennedy Krieger Institute — Baltimore
- University of Massachusetts — Worcester
- … и ещё 6 центров
Канада · 4 центра
- University of Calgary - Cumming School of Medicine — Calgary
- University of Alberta Hospital — Edmonton
- The Ottawa Hospital — Ottawa
- Genge Partners Inc — Montreal
Франция · 4 центра
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timon — Marseille
- CHU de Montpellier - Hopital Gui de Chauliac — Montpellier
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires — Nice
- AP-HP Hopital Pitie-Salpetriere — Paris
Германия · 4 центра
- Universitaetsklinikum Bonn — Bonn
- Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Go — Göttingen
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München
- Universitaetsklinikum Ulm — Ulm
Италия · 4 центра
- Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan
- Azienda Ospedaliero Universitaria Pisana — Pisa
- Azienda Ospedaliero-Universitaria Sant'Andrea — Roma
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuo — Roma
Япония · 4 центра
- National Hospital Organization Osaka Toneyama Medical Center — Osaka
- The University of Osaka Hospital — Osaka
- National Hospital Organization Sendai Nishitaga Hospital — Sendai
- National Center of Neurology and Psychiatry — Tokyo
Испания · 4 центра
- Hospital Universitari de Bellvitge — Barcelona
- Hospital Universitario Vall d'Hebron — Barcelona
- Hospital Universitario Donostia — San Sebastián
- Hospital Universitari i Politecnic La Fe — Valencia
Великобритания · 4 центра
- National Hospital for Neurology & Neurosurgery — London
- St. George's University Hospitals NHS Foundation Trust — London
- Royal Victoria Infirmary — Newcastle upon Tyne
- Royal Hallamshire Hospital — Sheffield
Дания · 2 центра
- Aarhus University Hospital — Aarhus
- Rigshospitalet — Copenhagen
Нидерланды · 2 центра
- Academisch Ziekenhuis Leiden — Leiden
- Stichting Radboud Universitair Medisch Centrum — Nijmegen
Идентификаторы
NCT: NCT07038200 · AOC 1020-CS3