Generalized Myasthenia Gravis, gMG, MG - Myasthenia Gravis
Conditions
Brief 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.
Interventions
BIOLOGICALEfgartigimod IV
Intravenous infusions of efgartigimod
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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: 1. intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) within 14 days of day 1 2. Rituximab within 6 months of day 1 3. Eculizumab within 1 month of day 1 4. Other monoclonal antibodies (eg, adalimumab, tocilizumab, ixekizumab)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean of the Average MG-ADL Total Score Change From Baseline During the Visit of Week 1 Through Week 21 by Regimen Arm | Up to 21 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of AEs, SAEs and AESIs | Up to 135 weeks | AE : adverse event; SAE: serious adverse event; AESI: adverse event of special interest |
| Change From Baseline in the MG-ADL Total Score Over Time | Up to 126 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
| Normalized Area Under the Effect Curve (AUEC) of MG-ADL Total Score Improvement From Baseline | Up to 21 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
| Characterization of MG-ADL Total Score Change From Baseline | Up to 21 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
| Percentage of Participants Who Have a ≥ 2, 3, 4, or 5 Points Improvement in MG-ADL Total Score From Baseline | Up to 21 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
| 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 | From week 4 to week 21 | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
| Percentage of Participants Who Achieve MSE, Defined as a MG-ADL Total Score of 0 or 1 | Up to 21 weeks | MSE = minimal symptom expression; The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment. |
Countries
Austria, Belgium, Canada, France, Georgia, Germany, Italy, Netherlands, Poland, Spain, United States
Participant flow
Recruitment details
The study was conducted at 31 sites that enrolled participants with anti-acetylcholine receptor antibody (AChR-Ab) seropositive gMG in 11 countries. A total of 98 participants were screened of whom 69 were randomized and treated. The results presented are based on the primary completion date. Remaining results will be reported within a year of global study completion.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 55.9 Age (years) STANDARD_DEVIATION 16.4 |
| Age, Customized Age, category 18 - 64 years | 44 Participants |
| Age, Customized Age, category >= 65 years | 25 Participants |
| Race/Ethnicity, Customized Ethnicity Hispanic or Latino | 0 Participants |
| Race/Ethnicity, Customized Ethnicity Not Hispanic or Latino | 50 Participants |
| Race/Ethnicity, Customized Race Asian | 1 Participants |
| Race/Ethnicity, Customized Race Black or African American | 2 Participants |
| Race/Ethnicity, Customized Race Other | 1 Participants |
| Race/Ethnicity, Customized Race Unknown | 1 Participants |
| Race/Ethnicity, Customized Race White | 64 Participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 17 | 0 / 52 |
| other Total, other adverse events | 16 / 17 | 43 / 52 |
| serious Total, serious adverse events | 1 / 17 | 7 / 52 |
Outcome results
None listed