An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Официальное название

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

Обзор

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Вмешательства

• Препарат Zilucoplan
  Zilucoplan will be administered subcutaneously to pediatric study participants

Первичные конечные точки

• Occurence of treatment emergent adverse events during the course of the study [Срок оценки: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
• Occurence of treatment-emergent serious adverse events (TESAEs) [Срок оценки: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
• Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product [Срок оценки: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
• Occurence of treatment-emergent infections [Срок оценки: Baseline (Day 1) to Safety Follow-up (up to Week 60)]

Вторичные конечные точки (5)

• Plasma concentration of Zilucoplan at Week 52 [Срок оценки: Week 52]
• Sheep red blood cell (sRBC) lysis activity at Week 52 [Срок оценки: Week 52]
• Blood complement component 5 (C5) levels at Week 52 [Срок оценки: Week 52]
• Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52 [Срок оценки: Week 52]
• Quantitative Myasthenia Gravis (QMG) score at Week 52 [Срок оценки: Week 52]

Критерии участия

Критерии включения

United States of America (USA) specific inclusion criterion:

\- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Rest of World (ROW) specific inclusion criterion:

\- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Global specific inclusion criteria:

• Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
• Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care

Критерии исключения

• Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
• Participant has known positive serology for muscle-specific kinase
• Participant has known hypersensitivity to any components of the IMP
• Participant has a prior history of meningococcal disease

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

Польша · 2 центра

• Mg0015 40774 — Katowice
• Mg0015 40218 — Warsaw

Великобритания · 2 центра

• Mg0015 40735 — Glasgow
• Mg0015 40736 — London

Италия · 1 центр

• Mg0015 40144 — Milan

South Korea · 1 центр

• Mg0015 20220 — Seoul

Идентификаторы

NCT: NCT06435312 · MG0015 · 2022-502073-42 · U1111-1290-3806

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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