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Recruiting 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

Phase II Interventional AChR-Ab Seropositive Generalized Myasthenia Gravis Myasthenia Gravis MG gMG

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Efgartigimod IV, Empasiprubart IV.
Who it may be relevant to
Registry conditions: AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis, MG, gMG. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Belgium, Germany, Greece, Italy +3
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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

Detailed 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.

Interventions

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

Primary outcome measures

  • Incidence of adverse events and serious adverse events in parts A and B [Time frame: Up to 21 weeks]
Secondary outcome measures (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 [Time frame: 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 [Time frame: Up to 21 weeks]
  • MG-ADL total score changes from baseline over time in part B compared with part A [Time frame: 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 [Time frame: Up to 18 weeks]
  • QMG total score changes from baseline over time in part B compared with part A [Time frame: Up to 21 weeks]
  • 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]
  • 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]

Eligibility criteria

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)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 11 centers
  • 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
  • … and 3 more centers
Italy · 3 centers
  • ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII — Bergamo
  • Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan
  • AORN Antonio Cardarelli — Naples
Poland · 3 centers
  • MICS Centrum Medyczne Bydgoszcz — Bydgoszcz
  • Centrum Medyczne Neurologia Slaska — Katowice
  • Warszawski Uniwersytet Medyczny — Warsaw
Greece · 2 centers
  • Eginitio Hospital — Athens
  • AHEPA University General Hospital of Thessaloniki — Thessaloniki
Spain · 2 centers
  • Hospital Regional Universitario de Malaga - Hospital General — Málaga
  • Hospital de La Santa Creu i Sant Pau — Barcelona
Belgium · 1 center
  • UZ Leuven - PPDS — Leuven
Germany · 1 center
  • Charité - Universitätsmedizin Berlin — Berlin
Japan · 1 center
  • IUHW Narita Hospital — Narita

Identifiers

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

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗