Phase 4 Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis

Официальное название

An Open-label, Prospective, Single-arm Study Assessing the Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis

Обзор

The purpose of the study is to assess the clinical efficacy of rozanolixizumab in adult Chinese participants with generalized myasthenia gravis (gMG) in the first Treatment Cycle.

Вмешательства

• Препарат Rozanolixizumab
  Subcutaneous infusion

Первичные конечные точки

• Change from Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score in the first Treatment Cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]

Вторичные конечные точки (7)

• MG-ADL responder (≥2.0-point improvement from Baseline) at Day 43 in the first Treatment Cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]
• Change from Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) score within first 6 week treatment cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]
• Change from Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) score within first 6-week treatment cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]
• Change from Baseline to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within first 6-week treatment cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]
• Change from Baseline to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within first 6-week treatment cycle [Срок оценки: Baseline to Day 43 (in first treatment cycle)]
• Percentage of participants with treatment-emergent adverse events (TEAEs) [Срок оценки: Up to End of study (40 weeks)]
• Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [Срок оценки: Up to End of study (40 weeks)]

Критерии участия

Критерии включения

• Study participant must be ≥18 years of age at the time of signing the informed consent form (ICF)
• Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at the Screening Visit based on study participant's history and supported by previous evaluation
• Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) documented in the medical history at the Screening Visit
• Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening Visit.
• Study participant with a myasthenia gravis-activities of daily living (MG-ADL) score of at least 3 points from non-ocular symptoms and a quantitative myasthenia gravis (QMG) score of at least 11 at the Screening and Baseline visits in the first Treatment Cycle.
• Study participant is considered for additional treatment by the investigator
• Body weight ≥35kg at the Screening Visit

Критерии исключения

• Study participant has a known hypersensitivity to any components of the study drug or any other anti-neonatal Fc receptor (anti-FcRn) medications
• Study participant has a clinically important active infection including unresolved or not adequately treated infection in the opinion of the investigator
• Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
• Study participant has previously received rozanolixizumab drug product
• Study participant has received any vaccine in the 4 weeks prior to the initiation of rozanolixizumab treatment or intends to receive any vaccine within 2 weeks after the last infusion of rozanolixizumab. Note: For participants that are on rozanolixizumab treatment, vaccination with live or live-attenuated vaccines is not recommended. During the whole study period, all other vaccines should take place at least 2 weeks after the last infusion of a Treatment Cycle and 4 weeks before initiating the next cycle
• Study participant has been treated with prohibited immunosuppressants, biologics, and other therapies within the timeframe shorter than the treatment-free period
• Study participant with severe (defined as Grade 3 on the MG-ADL scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis. Note: Impending Crisis is defined as bulbar or respiratory symptoms of a patient who significantly worsens in a short time (≤2 weeks) and meet MGFA IVb or score 3 in one QMG bulbar muscle item, or score 2 in respiratory muscle item, or bulbar+respiratory items score ≥4

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

Китай · 12 центров

• Mg0033 20040 — Пекин
• Mg0033 20261 — Changchun
• Mg0033 20295 — Чанша
• Mg0033 20348 — Фучжоу
• Mg0033 20269 — Гуанчжоу
• Mg0033 20185 — Цзинань
• Mg0033 20347 — Цзинань
• Mg0033 20172 — Шанхай
• … и ещё 4 центра

Идентификаторы

NCT: NCT07246564 · MG0033

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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