Phase 3
N=69
An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis · MG - Myasthenia Gravis · gMG
Bottom Line
View on ClinicalTrials.gov: NCT04980495 ↗Enrolled (actual)
69
Serious AEs
11.6%
Results posted
May 2026
Primary outcome: Primary: Mean of the Average MG-ADL Total Score Change From Baseline During the Visit of Week 1 Through Week 21 by Regimen Arm — -5.13; -4.61 Score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Efgartigimod IV (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- argenx
- Primary completion
- Aug 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean of the Average MG-ADL Total Score Change From Baseline During the Visit of Week 1 Through Week 21 by Regimen Arm |
-5.13; -4.61 | — |
| SECONDARY Incidence of AEs, SAEs and AESIs |
— | — |
| SECONDARY Change From Baseline in the MG-ADL Total Score Over Time |
— | — |
| SECONDARY Normalized Area Under the Effect Curve (AUEC) of MG-ADL Total Score Improvement From Baseline |
— | — |
| SECONDARY Characterization of MG-ADL Total Score Change From Baseline |
— | — |
| SECONDARY Percentage of Participants Who Have a ≥ 2, 3, 4, or 5 Points Improvement in MG-ADL Total Score From Baseline |
— | — |
| SECONDARY Percentage of Time Participants Have a Change in MG-ADL Total Score of at Least 2 Points From Baseline During Week 4 Through Week 21 |
— | — |
| SECONDARY Percentage of Participants Who Achieve MSE, Defined as a MG-ADL Total Score of 0 or 1 |
— | — |
Summary
The purpose of this open-label study is to investigate the efficacy, safety, and tolerability of a continuous regimen of efgartigimod compared with a cyclic regimen in participants with Generalized Myasthenia Gravis (gMG).
Participants will receive efgartigimod throughout the study. The participants will be randomized to the continuous regimen arm or to the cyclic regimen arm. The study consists of a part A (regimen comparison period) where participants will continue the treatment based on the treatment regimen arm they were assigned at randomization. Following part A, participants will enter part B (extension period) where all participants will receive efgartigimod in the continuous regimen. The study duration for participants is up to 138 weeks.
Eligibility Criteria
Inclusion Criteria
- At least 18 years of age, at the time of signing the informed consent.
- Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by a physical exam and confirmed seropositivity for anti-acetylcholine receptor antibodies (AChR-Abs).
- Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, or IV
Exclusion Criteria
- Any other condition that, in the opinion of the investigator, would interfere with an accurate assessment of the clinical symptoms of gMG and/or put the participant at undue risk
- A thymectomy within 3 months of screening
- Use of the following prior or concomitant therapies:
- intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) within 14 days of day 1
- Rituximab within 6 months of day 1
- Eculizumab within 1 month of day 1
- Other monoclonal antibodies (eg, adalimumab, tocilizumab, ixekizumab)
Data sourced from ClinicalTrials.gov (NCT04980495). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.