ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis

Официальное название

ISA2 to Master Protocol ARGX-999-2-MG-2000 - an Exploratory, Phase 2a, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis

Обзор

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life. The aim of this ISA2 is to investigate the effects of empasiprubart in participants with AChR-Ab seropositive generalized myasthenia gravis (gMG). The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170 More information can be found here: https://clinicaltrials.argenx.com/adaptforward2

Подробное описание

Once the master protocol and ISA2 screening periods are completed, eligible participants will be randomized to receive empasiprubart IV or placebo in the double-blinded treatment period (DBTP). All participants will then receive open-label efgartigimod PH20 SC PFS in the safety follow-up period.

The study duration for each participant is approximately up to 45 weeks.

Вмешательства

• Биопрепарат Empasiprubart IV
  Intravenous infusions of empasiprubart
• Другое Placebo IV
  Intravenous infusions of placebo
• Комбинированный продукт Efgartigimod PH20 SC PFS
  Subcutaneous administration of efgartigimod PH20 via pre-filled syringe (PFS)

Первичные конечные точки

• Incidence of adverse events and serious adverse events in the DBTP [Срок оценки: Up to 12 weeks]

Вторичные конечные точки (9)

• MG-ADL total score change from baseline at week 12 [Срок оценки: Up to 12 weeks]
• QMG total score change from baseline at week 12 [Срок оценки: Up to 12 weeks]
• MG-ADL total score change from baseline over time up to week 12 [Срок оценки: Up to 12 weeks]
• QMG total score change from baseline over time up to week 12 [Срок оценки: Up to 12 weeks]
• Proportion of participants reaching MSE at any point by week 12 [Срок оценки: Up to 12 weeks]
• Proportion of participants who have ≥3-point reduction in MG-ADL at week 12 [Срок оценки: Up to 12 weeks]
• Proportion of participants who have ≥5-point reduction in QMG at week 12 [Срок оценки: Up to 12 weeks]
• Proportion of participants who have a positive PASS at week 12 [Срок оценки: Up to 12 weeks]
• Proportion of participants who have a 50% MG-ADL total score improvement at week 12 [Срок оценки: Up to 12 weeks]

Критерии участия

Критерии включения

• Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab).
• Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb.
• Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years before ISA screening or will complete immunization at least 14 days before the first IMP administration.

Критерии исключения

• Clinical diagnosis of systemic lupus erythematosus (SLE).
• Is receiving concurrent complement inhibitors (eg, eculizumab, zilucoplan, ravulizumab, or others). Participants who received zilucoplan or eculizumab >2 months or ravulizumab >6 months before baseline are allowed to participate.
• Has received an FcRn antagonist, including efgartigimod, within 4 weeks before baseline.
• Had prior empasiprubart exposure.

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 · 2 центра

• Visionary Investigators Network — Miami
• Erlanger Health System — Chattanooga

Идентификаторы

NCT: NCT07673627 · ARGX-999-2-MG-20002 · 2025-522939-33-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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