AChR-Ab Seropositive Generalized Myasthenia Gravis, Generalized Myasthenia Gravis, Generalized Myasthenia Gravis (gMG), gMG, MG, Myasthenia Gravis
Conditions
Brief summary
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
BIOLOGICALEfgartigimod IV
Intravenous infusion of efgartigimod
BIOLOGICALEmpasiprubart IV
Intravenous infusion of empasiprubart
Sponsors
argenx
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events and serious adverse events in parts A and B | Up to 21 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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) |
| Proportion of participants reaching MSE at any point in part B cycles 1 and 2, and part B cycles 1 or 2 | Up to 21 weeks | MSE: Minimal symptom expression |
| MG-ADL total score changes from baseline over time in part B compared with part A | 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) |
| 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 | 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) |
| QMG total score changes from baseline over time in part B compared with part A | 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) |
| Proportion of participants who have a 50% MG ADL total score improvement in part B cycles 1 and 2 | 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) |
| Proportion of participants who have positive PASS in part B cycles 1 and 2, and part B cycles 1 or 2 | Up to 21 weeks | PASS: Patient acceptable symptom state |
Countries
Belgium, Germany, Greece, Italy, Japan, Poland, Spain, United States
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed