Adding empasiprubart to efgartigimod for AChR-antibody positive generalized myasthenia gravis
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
This trial will test whether adding empasiprubart to efgartigimod infusions helps people with AChR-antibody positive generalized myasthenia gravis who have only had a partial response to efgartigimod.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 70 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | argenx Industry-sponsored |
| Drugs / interventions | eculizumab, ravulizumab |
| Locations | 9 sites (Carlsbad, California and 8 other locations) |
| Trial ID | NCT07284420 on ClinicalTrials.gov |
What this trial studies
This Phase 2 add-on study enrolls AChR-Ab seropositive participants with generalized myasthenia gravis who complete a run-in period of efgartigimod IV (part A). Eligible participants then enter an add-on period (part B) receiving both efgartigimod IV and empasiprubart IV, while those not eligible proceed to a safety follow-up (part C) receiving efgartigimod alone. The trial is conducted under the ADAPT Forward platform protocol and includes screening, treatment, and follow-up visits over roughly 54 weeks per participant. All participants must meet vaccination requirements for encapsulated bacteria or be willing to be vaccinated before dosing.
Who should consider this trial
Good fit: Adults with AChR-Ab seropositive generalized myasthenia gravis (MGFA class II–IV) who have shown a partial clinical response to efgartigimod and who are vaccinated or willing to be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae.
Not a fit: People with systemic lupus erythematosus, known complement deficiencies, recent use of complement inhibitors (within protocol-specified windows), or those proven refractory to efgartigimod are unlikely to receive benefit from this add-on approach.
Why it matters
Potential benefit: If successful, adding empasiprubart could improve symptom control for people who only partially respond to efgartigimod and potentially reduce the need for other immunosuppressive treatments.
How similar studies have performed: Efgartigimod has demonstrated clinical benefit in gMG in prior studies, while empasiprubart as an add-on therapy represents a newer approach with limited published data to date.
Eligibility criteria
Show full inclusion / exclusion 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)
Where this trial is running
Carlsbad, California and 8 other locations
- Profound Research LLC - Carlsbad — Carlsbad, California, United States (Recruiting)
- Visionary Investigators Network — Miami, Florida, United States (Recruiting)
- Dent Neurologic Institute - Amherst — Amherst, New York, United States (Recruiting)
- Erlanger Health System — Chattanooga, Tennessee, 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)
- 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
- Email: clinicaltrials@argenx.com
- Phone: 857-350-4834
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.