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

argenx

Status and phase: Enrolling; Phase 2

Conditions: MG; Myasthenia Gravis; AChR-Ab Seropositive Generalized Myasthenia Gravis; Generalized Myasthenia Gravis (gMG); Generalized Myasthenia Gravis; gMG

Treatments: Biological: Efgartigimod IV; Biological: Empasiprubart IV

Study type: Interventional

Funder types: Industry

Identifiers

NCT07284420

ARGX-999-2-MG-20001

2025-522492-28-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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

Full description

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.

Enrollment: 70 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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70 participants in 2 patient groups

Efgartigimod IV + Empasiprubart IV

Experimental group

Description:

Participants receive efgartigimod IV in part A, B and C and empasiprubart IV in part B

Treatment:

Biological: Empasiprubart IV

Biological: Efgartigimod IV

Efgartigimod IV (part A + C)

Experimental group

Description:

Participants not eligible for part B, receiving efgartigimod IV in part A and C

Treatment:

Biological: Efgartigimod IV