New Onset Generalized Myasthenia Gravis (gMG)
Conditions
Brief summary
The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.
Interventions
BIOLOGICALEfgartigimod PH20 SC
Subcutaneous injection of efgartigimod PH20 SC
Sponsors
argenx
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Is at least 18 years when signing the ICF * Has been diagnosed with gMG of MGFA class II, III, or IV * Is seropositive for AChR-Ab * Is treatment-naive for gMG or has been administered AChEI for the treatment of gMG * Had onset of generalized MG signs and/or symptoms within 12 months before screening; candidates who also had onset of ocular MG signs and/or symptoms within 24 months before screening may be enrolled in the study * Has an MG-ADL score ≥5
Exclusion criteria
* gMG diagnosis of MGFA class I or V * Underwent a thymectomy prior to screening, except thymectomy for treatment of nonmalignant thymoma prior to the gMG diagnosis * Prior or current use of any of any systemic corticosteroid therapy or nonsteroidal immunosuppressive therapy for the treatment of gMG
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants who achieve MSE of MG at any time within the first 16 weeks of treatment with efgartigimod PH20 SC | up to 16 weeks | MSE (minimal symptom expression) is defined as an MG-ADL (Myasthenia Gravis Activities of Daily Living) total score of 0 or 1. The MG-ADL scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes from baseline in MG-ADL total scores over time | up to 51 weeks | The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe). |
| Changes from baseline in MG-QOL-15r total scores over time | up to 51 weeks | The MG-QoL15r (Myasthenia Gravis Quality of Life 15-item scale revised) questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life). |
| Proportion of participants who are administered systemic corticosteroid therapy as background add-on therapy throughout the study | up to 55 weeks | — |
| Incidence of adverse events, serious adverse events and adverse events leading to study drug discontinuation | up to 55 weeks | — |
Countries
United States
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed