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Recruiting NCT07673627

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

Phase II Interventional AChR-Ab Seropositive Generalized Myasthenia Gravis Myasthenia Gravis (MG) MG - Myasthenia Gravis 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: Empasiprubart IV, Placebo IV, Efgartigimod PH20 SC PFS.
Who it may be relevant to
Registry conditions: AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis (MG), MG - Myasthenia Gravis, 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
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

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

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 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

Detailed description

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.

Interventions

  • Biological Empasiprubart IV
    Intravenous infusions of empasiprubart
  • Other Placebo IV
    Intravenous infusions of placebo
  • Combination product Efgartigimod PH20 SC PFS
    Subcutaneous administration of efgartigimod PH20 via pre-filled syringe (PFS)

Primary outcome measures

  • Incidence of adverse events and serious adverse events in the DBTP [Time frame: Up to 12 weeks]
Secondary outcome measures (9)
  • MG-ADL total score change from baseline at week 12 [Time frame: Up to 12 weeks]
  • QMG total score change from baseline at week 12 [Time frame: Up to 12 weeks]
  • MG-ADL total score change from baseline over time up to week 12 [Time frame: Up to 12 weeks]
  • QMG total score change from baseline over time up to week 12 [Time frame: Up to 12 weeks]
  • Proportion of participants reaching MSE at any point by week 12 [Time frame: Up to 12 weeks]
  • Proportion of participants who have ≥3-point reduction in MG-ADL at week 12 [Time frame: Up to 12 weeks]
  • Proportion of participants who have ≥5-point reduction in QMG at week 12 [Time frame: Up to 12 weeks]
  • Proportion of participants who have a positive PASS at week 12 [Time frame: Up to 12 weeks]
  • Proportion of participants who have a 50% MG-ADL total score improvement at week 12 [Time frame: Up to 12 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 before ISA screening or will complete immunization at least 14 days before the first IMP administration.

Exclusion criteria

  • 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.

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

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 2 centers
  • Visionary Investigators Network — Miami
  • Erlanger Health System — Chattanooga

Identifiers

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

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗