ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

Официальное название

An ISA to Master Protocol ARGX-999-2-MG-2000 for an Exploratory, Phase 2a, Proof-of-Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart IV as Add-On Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

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 ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis. The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170 More information can be found here: https://clinicaltrials.argenx.com/adaptforward1

Подробное описание

Once the master protocol and ISA1 screening periods are completed, eligible participants can enroll in the run-in period (part A) where they will receive efgartigimod IV. Eligible participants can then continue to the add-on period (part B) where they will receive both efgartigimod IV and empasiprubart IV.

Participants who are not eligible for part B will continue directly to the safety follow-up period (part C) where they will receive efgartigimod IV only.

The study duration for each participant is approximately up to 54 weeks.

Вмешательства

• Биопрепарат Efgartigimod IV
  Intravenous infusion of efgartigimod
• Биопрепарат Empasiprubart IV
  Intravenous infusion of empasiprubart

Первичные конечные точки

• Incidence of adverse events and serious adverse events in parts A and B [Срок оценки: Up to 21 weeks]

Вторичные конечные точки (7)

• MG-ADL total score change from baseline at week 18 in part B (cycle 2 day 29) compared with MG ADL total score change from baseline at week 4 in part A [Срок оценки: Up to 18 weeks]
• Proportion of participants reaching MSE at any point in part B cycles 1 and 2, and part B cycles 1 or 2 [Срок оценки: Up to 21 weeks]
• MG-ADL total score changes from baseline over time in part B compared with part A [Срок оценки: Up to 21 weeks]
• QMG total score change from baseline at week 18 in part B (cycle 2 day 29) compared with QMG total score change from baseline at week 4 in part A [Срок оценки: Up to 18 weeks]
• QMG total score changes from baseline over time in part B compared with part A [Срок оценки: Up to 21 weeks]
• Proportion of participants who have a 50% MG ADL total score improvement in part B cycles 1 and 2 [Срок оценки: Up to 21 weeks]
• Proportion of participants who have positive PASS in part B cycles 1 and 2, and part B cycles 1 or 2 [Срок оценки: Up to 21 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 of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration

Критерии исключения

• Clinical diagnosis of systemic lupus erythematosus (SLE)
• Any known complement deficiency
• Current administration of a complement inhibitor or received zilucoplan or eculizumab <2 months or ravulizumab <6 months before the first study drug administration
• Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points)

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

США · 11 центров

• Profound Research LLC - Carlsbad — Carlsbad
• Visionary Investigators Network — Miami
• University of South Florida — Tampa
• Massachusetts General Hospital — Boston
• Henry Ford Health Systems — Detroit
• Dent Neurologic Institute - Amherst — Amherst
• Duke Early Phase Clinical Research Unit - PPDS — Durham
• Ohio State Martha Morehouse Outpatient Care — Columbus
• … и ещё 3 центра

Италия · 3 центра

• ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII — Bergamo
• Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan
• AORN Antonio Cardarelli — Naples

Польша · 3 центра

• MICS Centrum Medyczne Bydgoszcz — Bydgoszcz
• Centrum Medyczne Neurologia Slaska — Katowice
• Warszawski Uniwersytet Medyczny — Warsaw

Греция · 2 центра

• Eginitio Hospital — Athens
• AHEPA University General Hospital of Thessaloniki — Thessaloniki

Испания · 2 центра

• Hospital Regional Universitario de Malaga - Hospital General — Málaga
• Hospital de La Santa Creu i Sant Pau — Barcelona

Бельгия · 1 центр

• UZ Leuven - PPDS — Leuven

Германия · 1 центр

• Charité - Universitätsmedizin Berlin — Berlin

Япония · 1 центр

• IUHW Narita Hospital — Narita

Идентификаторы

NCT: NCT07284420 · ARGX-999-2-MG-20001 · 2025-522492-28-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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