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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522939-33-00 | EU Trial (CTIS) Number |
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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
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Empasiprubart IV | Experimental | Participants receive empasiprubart IV in the DBTP |
|
| Placebo IV | Placebo Comparator | Participants receive placebo IV in the DBTP |
|
| Efgartigimod PH20 SC PFS | Other | Participants receive open-label efgartigimod PH20 SC PFS in the safety follow-up period |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Empasiprubart IV | Biological | Intravenous infusions of empasiprubart |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events and serious adverse events in the DBTP | Up to 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | Up to 12 weeks |
| QMG total score change from baseline at week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sabine Coppieters, MD | Contact | 857-350-4834 | clinicaltrials@argenx.com |
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| Placebo IV |
| Other |
Intravenous infusions of placebo |
|
| Efgartigimod PH20 SC PFS | Combination Product | Subcutaneous administration of efgartigimod PH20 via pre-filled syringe (PFS) |
|
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) |
| 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) | 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) | Up to 12 weeks |
| Proportion of participants reaching MSE at any point by week 12 | MSE: Minimal symptom expression | 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). | 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). | Up to 12 weeks |
| Proportion of participants who have a positive PASS at week 12 | PASS: Patient acceptable symptom state | 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). | Up to 12 weeks |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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