ADAPT Forward master protocol testing multiple regimens for generalized myasthenia gravis

A Master Protocol for an Exploratory, Phase 2a, Proof-of-Concept Platform Study to Evaluate the Safety, Tolerability, and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis

This platform tests several drug regimens, including efgartigimod IV and empasiprubart IV, in adults with generalized myasthenia gravis to find safer and more effective treatment combinations.

Quick facts

• Study type: Observational
• Enrollment: 70 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: argenx (industry)
• Locations: 10 sites (Carlsbad, California and 9 other locations)
• Trial ID: NCT07294170 on ClinicalTrials.gov

What this trial studies

ADAPT Forward uses a single master protocol that lets multiple intervention-specific appendices (ISAs) run under the same overall framework. The design includes a common master screening period, ISA-specific screening, an ISA treatment period of variable duration and design, and ISA safety follow-up periods. The initial ISA will test empasiprubart IV as an add-on to efgartigimod IV in AChR-antibody positive participants with a partial response to efgartigimod. Sites in California, Florida, and Maryland will follow ISA-specific procedures for enrollment, treatment, and follow-up.

Who should consider this trial

Good fit: Adults (at least 18 years old) with clinician-diagnosed generalized myasthenia gravis who are stable on background MG therapies are the intended participants.

Not a fit: People with other autoimmune diseases or unstable medical conditions, or those who are MGFA Class V, are unlikely to benefit or be eligible for participation.

Why it matters

Potential benefit: If successful, the program could identify treatment combinations that reduce symptoms and side effects and improve quality of life for people with generalized myasthenia gravis.

How similar studies have performed: FcRn-targeting agents such as efgartigimod have shown benefit in gMG in prior trials, but using empasiprubart as an add-on within this platform is a relatively novel approach.

Eligibility criteria

Inclusion Criteria:

* Is at least 18 years of age and the local legal age of consent for clinical studies
* Has been diagnosed with MG with consistent clinical features per the investigator's clinical judgment
* If receiving MG therapy, including nonsteroidal immunosuppressive drugs (NSIDs), corticosteroids, or acetylcholinesterase (AChE) inhibitors either in combination or alone, the participant should receive a stable dosage before master protocol screening

Exclusion Criteria:

* Known autoimmune disease or any medical condition other than the indication under study that would interfere with an accurate assessment of clinical symptoms of MG or puts the participant at undue risk
* Is MGFA (Myasthenia Gravis Foundation of America) Class V

Where this trial is running

Carlsbad, California and 9 other locations

• Profound Research LLC - Carlsbad — Carlsbad, California, United States (RECRUITING)
• Visionary Investigators Network — Miami, Florida, United States (RECRUITING)
• Erlanger Health System — Columbia, Maryland, United States (RECRUITING)
• Dent Neurologic Institute - Amherst — Amherst, New York, United States (RECRUITING)
• National Neuromuscular Research Institute — Austin, Texas, United States (RECRUITING)
• UZ Leuven - PPDS — Leuven, Belgium (RECRUITING)
• MICS Centrum Medyczne Bydgoszcz — Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland (RECRUITING)
• Krakowska Akademia Neurologii Sp. z o.o. — Krakow, Lesser Poland Voivodeship, Poland (RECRUITING)
• Centrum Medyczne Neurologia Slaska — Katowice, Silesian Voivodeship, Poland (RECRUITING)
• Hospital Regional Universitario de Malaga - Hospital General — Málaga, Malaga, Spain (RECRUITING)

Study contacts

• Study coordinator: Sabine Coppieters, MD

Conditions: Myasthenia Gravis, MG, gMG, Generalized Myasthenia Gravis, AChR-Ab Seropositive Generalized Myasthenia Gravis