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
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
40
Intravenous infusions of empasiprubart
Intravenous infusions of placebo
Subcutaneous administration of efgartigimod PH20 via pre-filled syringe (PFS)
Visionary Investigators Network
Miami, Florida, United States
RECRUITINGDent Neurologic Institute - Amherst
Amherst, New York, United States
RECRUITINGErlanger Health System
Chattanooga, Tennessee, United States
RECRUITINGNational Neuromuscular Research Institute
Austin, Texas, United States
ACTIVE_NOT_RECRUITINGIncidence of adverse events and serious adverse events in the DBTP
Time frame: Up to 12 weeks
MG-ADL total score change from baseline at week 12
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)
Time frame: Up to 12 weeks
QMG total score change from baseline at week 12
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)
Time frame: Up to 12 weeks
MG-ADL total score change from baseline over time up to week 12
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)
Time frame: Up to 12 weeks
QMG total score change from baseline over time up to week 12
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)
Time frame: Up to 12 weeks
Proportion of participants reaching MSE at any point by week 12
MSE: Minimal symptom expression
Time frame: Up to 12 weeks
Proportion of participants who have ≥3-point reduction in MG-ADL at week 12
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).
Time frame: Up to 12 weeks
Proportion of participants who have ≥5-point reduction in QMG at week 12
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).
Time frame: Up to 12 weeks
Proportion of participants who have a positive PASS at week 12
PASS: Patient acceptable symptom state
Time frame: Up to 12 weeks
Proportion of participants who have a 50% MG-ADL total score improvement at week 12
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).
Time frame: Up to 12 weeks
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