Skip to main content
OpenTrials
Recruiting

NCT Number: NCT07284420

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

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

Recruiting

Interested in participating?

Request Info

Key information

Conditions

AChR-Ab Seropositive Generalized Myasthenia Gravis Autoimmune Diseases Autoimmune Diseases of the Nervous System Generalized Myasthenia Gravis Generalized Myasthenia Gravis (gMG) Immune System Diseases MG Myasthenia Gravis Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Junction Diseases Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System gMG

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UZ Leuven - PPDS, Leuven, Belgium

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

efgartigimod IV

Biological

Intravenous infusion of efgartigimod

Empasiprubart IV

Biological

Intravenous infusion of empasiprubart

Primary outcomes

  1. Incidence of adverse events and serious adverse events in parts A and B

    Time frame: Up to 21 weeks

Secondary outcomes

  1. 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

    Time frame: Up to 18 weeks

    The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

  2. Proportion of participants reaching MSE at any point in part B cycles 1 and 2, and part B cycles 1 or 2

    Time frame: Up to 21 weeks

    MSE: Minimal symptom expression

  3. MG-ADL total score changes from baseline over time in part B compared with part A

    Time frame: Up to 21 weeks

    The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

  4. 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

    Time frame: Up to 18 weeks

    The Quantitative myasthenia gravis (QMG) includes 13 items that measure endurance or fatigability and accounts for fluctuations in disease state. The total score ranges from 0 (no disease severity) to 39 (highest disease severity)

  5. QMG total score changes from baseline over time in part B compared with part A

    Time frame: Up to 21 weeks

    The Quantitative myasthenia gravis (QMG) includes 13 items that measure endurance or fatigability and accounts for fluctuations in disease state. The total score ranges from 0 (no disease severity) to 39 (highest disease severity)

  6. Proportion of participants who have a 50% MG ADL total score improvement in part B cycles 1 and 2

    Time frame: Up to 21 weeks

    The Myasthenia Gravis Activities of Daily Living (MG ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

  7. Proportion of participants who have positive PASS in part B cycles 1 and 2, and part B cycles 1 or 2

    Time frame: Up to 21 weeks

    PASS: Patient acceptable symptom state

Study contacts

Contact information is provided by the study sponsor or research team.

Sabine Coppieters, MD

CONTACT

[email protected]

857-350-4834

Sponsors and collaborators

Lead sponsor

argenx

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Dec 16, 2025
Registry last updated
Jul 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.