AChR-Ab Seropositive Generalized Myasthenia Gravis, gMG, MG - Myasthenia Gravis, Myasthenia Gravis (MG)
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 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
BIOLOGICALEmpasiprubart IV
Intravenous infusions of empasiprubart
OTHERPlacebo IV
Intravenous infusions of placebo
COMBINATION_PRODUCTEfgartigimod PH20 SC PFS
Subcutaneous administration of efgartigimod PH20 via pre-filled syringe (PFS)
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
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 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events and serious adverse events in the DBTP | Up to 12 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| MG-ADL total score change from baseline at week 12 | Up to 12 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 12 | Up to 12 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) |
| MG-ADL total score change from baseline over time up to week 12 | Up to 12 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 over time up to week 12 | Up to 12 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 reaching MSE at any point by week 12 | Up to 12 weeks | MSE: Minimal symptom expression |
| Proportion of participants who have ≥3-point reduction in MG-ADL at week 12 | Up to 12 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 ≥5-point reduction in QMG at week 12 | Up to 12 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 positive PASS at week 12 | Up to 12 weeks | PASS: Patient acceptable symptom state |
| Proportion of participants who have a 50% MG-ADL total score improvement at week 12 | Up to 12 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). |
Countries
United States
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed