A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)
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: Telitacicept, Placebo.
- Кому может быть актуально
- Состояния в реестре: Generalized Myasthenia Gravis. Базовые параметры: от 18 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, Аргентина, Австралия, Бельгия, Бразилия +9
- 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 Study With an Open-label Extension Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis
Обзор
The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.
Подробное описание
Myasthenia gravis (MG) is an autoimmune disease in which autoantibodies disrupt the postsynaptic membrane, impairing nerve-to-muscle signal transmission. The predominant manifestation is muscle weakness, which typically worsens with repeated muscle exertion, such that function is usually the best in the morning with more pronounced weakness at the end of the day. A major challenge in MG is the lack of therapies that effectively treat the disease.
Telitacicept is a fully human TACI-Fc fusion protein that targets B-lymphocyte stimulator (BLyS) and A proliferating-inducing ligand (APRIL), neutralizing their interactions with receptors on B cells. The blockage of BLyS and APRIL interaction with their respective cell membrane receptors (transmembrane activator and CAML interactor \[TACI\], B-cell maturation antigen, and BLyS receptors) by telitacicept can inhibit B-cell proliferation and maturation. This suppression at the proximal portion of the immune response could alleviate autoimmune symptoms.
This is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension (OLE) to evaluate the efficacy and safety of telitacicept in a global patient population with gMG. The total duration of the study is variable but will include an approximately 4-week screening period, a 24-week double-blind treatment period (Week 0 through Week 24), a 48-week OLE (Week 24 through Week 72), followed by an extended OLE period (E-OLE), and an 8-week end-of-study follow-up period. The E-OLE is variable duration, defined as the period after the 48-week OLE period until telitacicept is approved for MG in the country or the further development in the indication is concluded.
Вмешательства
- Биопрепарат Telitacicept
Subcutaneous injection - Препарат Placebo
Subcutaneous injection
Первичные конечные точки
- Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24 [Срок оценки: Week 24]
Вторичные конечные точки (5)
- Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24 [Срок оценки: Week 24]
- Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24 [Срок оценки: Week 24]
- Proportion of patients with a decrease of ≥2 points from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24 [Срок оценки: Week 24]
- Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24 [Срок оценки: Week 24]
- Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24 [Срок оценки: Week 24]
Критерии участия
Критерии включения
- Male or female patient aged ≥18 years at screening.
- Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV.
- Patients have positive antibodies against AChR or MuSK at screening.
- MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.
- QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline.
Критерии исключения
- Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk.
- Patients having acute or chronic infection.
- Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening. Patients with thymoma diagnosed 3-5 years prior to screening may be eligible if thymoma was at a localized stage and definitively treated with complete surgical resection.
- Patients having current or history of primary immunodeficiency.
- Patients having history of malignancy within the last 5 years.
- Patient having prior or continuing diagnosis of serious cardiovascular, liver, kidney, respiratory system, endocrine or hematologic disease.
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 · 26 центров
- HonorHealth Neurology - Bob Bové Neuroscience Institute — Scottsdale
- University of Southern California — Los Angeles
- University of California Irvine — Orange
- University of California San Francisco (UCSF) — San Francisco
- SFM Clinical Research — Boca Raton
- Allied Biomedical Research Institute (ABRI) — Miami
- Medsol Clinical Research Center — Port Charlotte
- University of South Florida Health (USF Health) — Tampa
- … и ещё 18 центров
Китай · 19 центров
- AnHui Provincial Hospital — Хэфэй
- Beijing Hospital — Пекин
- The First Affiliated Hospital of Chongqing Medical University — Чунцин
- Fujian Medical University Union Hospital — Фучжоу
- The Second Hospital & Clinical Medical School. Lan Zhou University — Lanzhou
- The First Affiliated Hospital, Sun Yat-Sen University — Гуанчжоу
- Affiliated Hospital of ZunYi Medical University — Zunyi
- TongJi Medical College of HUST — Ухань
- … и ещё 11 центров
Япония · 16 центров
- National Hospital Organization - Nagoya Medical Center — Nagoya
- … и ещё 15 центров
Польша · 11 центров
Список центров уточняется — проверьте первичный протокол.
Италия · 8 центров
- IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo — San Giovanni Rotondo
- Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli — Naples
- Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli — Naples
- Azienda Ospedaliera Universitaria Federico II — Naples
- Fondazione Istituto San Raffaele - G. Giglio Di Cefalu — Cefalù
- Istituto Neurologico Casimiro Mondino — Pavia
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome
- Azienda Ospedaliera - Universitaria Sant' Andrea — Rome
Аргентина · 5 центров
- STAT Research S.A. — Ciudad Autónoma de Buenos Aires
- Hospital Italiano de Buenos Aires — Ciudad Autónoma de Buenos Aires
- Hospital General de Agudos José María Ramos Mejía — Ciudad Autónoma de Buenos Aires
- Fundación Scherbovsky — Mendoza
- INECO Neurociencias Oroño — Rosario
Бразилия · 5 центров
- Instituto de Neurologia de Curitiba — Curitiba
- Hospital São Lucas da PUCRS — Porto Alegre
- Universidade Estadual de Campinas — Campinas
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto — Ribeirão Preto
- Pseg Centro de Pesquisa Clinica — São Paulo
Испания · 5 центров
Список центров уточняется — проверьте первичный протокол.
Грузия · 4 центра
- Multi-profile Clinic "New Hospitals" — Tbilisi
- Pineo Medical Ecosystem — Tbilisi
- Aversi Clinic - Central Branch — Tbilisi
- Simon Khechinashvili University Hospital — Tbilisi
Австралия · 3 центра
- Concord Repatriation General Hospital — Concord
- Townsville University Hospital — Douglas
- Gold Coast University Hospital — Southport
Чехия · 3 центра
- Fakultni Nemocnice Ostrava — Ostrava-Poruba
- MINKSneuro s.r.o. - MUDr. Eduard Minks Ph.D. — Brno
- Fakultni Nemocnice Brno — Brno
Бельгия · 2 центра
- Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg — Leuven
- Centre Hospitalier Régional de la Citadelle — Liège
Канада · 2 центра
- University Hospital - London Health Sciences Centre — London
- Toronto General Hospital — Toronto
Франция · 2 центра
- Hôpital Pasteur — Nice
- Groupe Hospitalier Pellegrin — Bordeaux
Идентификаторы
NCT: NCT06456580 · RC18G006