Generalized Myasthenia Gravis, gMG
Conditions
Keywords
Generalized myasthenia gravis, gMG, Glucocorticoid toxicity, Complement C5 inhibitor, Prednisone reduction, Cortisol monitoring, ravulizumab, corticosteroids, adrenal insufficiency, steroid sparing
Brief summary
This is a prospective, multicenter single arm study designed to evaluate the effectiveness and safety of a predefined oral corticosteroids (OCS) tapering schedule to reduce OCS use in adult participants with acetylcholine receptor positive (AChR+) generalized myasthenia gravis (gMG) being treated with intravenous ravulizumab.
Interventions
PROCEDUREOral Corticosteroid Tapering Schedule
During the tapering period, participant will follow an Oral Corticosteroid tapering schedule as applicable to the participant. The oral corticosteroid (prednisone/prednisolone) is provided as per standard medical care.
DRUGRavulizumab
Patients being treated with intravenous ravulizumab as part of their standard medical care.
Participants will continue with or be switched to prednisone/prednisolone as per standard medical care.
Sponsors
Alexion Pharmaceuticals, Inc.
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
* Aged greater than 18 years and male or female * Clinical diagnosis of gMG * Receiving ravulizumab treatment prior to enrollment * Receipt of OCS therapy equivalent to a daily dose ≥ 7.5 mg of prednisone/prednisolone for ≥ 4 continuous weeks directly preceding enrollment * A participant of childbearing potential must have a negative highly sensitive pregnancy test (serum or urine, as required by local regulations) taken at Screening before OCS tapering begins. * Willing to sign informed consent
Exclusion criteria
* Concurrent participation in an interventional clinical trial. * History of chronic hypoadrenalism (ie, Addison's disease). * Use of concomitant OCS for comorbid conditions other than gMG * Receipt of a biologic for gMG (e.g., efgartigimod, rozanolixizumab, inebilizumab, rituximab, intravenous immunoglobulin) within 5 half-lives of enrollment * Pregnant, breastfeeding, or intending to conceive during the course of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of Adult Participants With gMG Who Either: Discontinue Oral Corticosteroids (OCS) or Reduce Their Daily OCS Dosage ≤ 5 mg/day and Maintain this Status for ≥ 4 Weeks Without Clinical Deterioration of gMG | Up to approximately 32 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of Adult Participants with gMG Who Discontinue OCS (0 mg/day), Sustained ≥ 4 Weeks Without Clinical Deterioration of gMG | Up to approximately 32 weeks |
| Percentage Change From Daily OCS Dose (mg/day) at Baseline | Baseline, Up to approximately 32 weeks |
| Change from Baseline in the Myasthenia Gravis Quality of Life-15 Revised (MG-QoL-15r) Score in Adult Participants with gMG | Baseline, Up to approximately 32 weeks |
| Change from Baseline in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score in Adult Participants with gMG | Baseline, Up to approximately 32 weeks |
| Cumulative Worsening Score per the Glucocorticoid Toxicity Index - Metabolic Domains (GTI-MD) in Adult Participants with gMG | Baseline up to approximately 32 weeks |
| Aggregate Improvement Score per the GTI-MD in Adult Participants with gMG | Baseline up to approximately 32 weeks |
Countries
Germany, Italy, Japan, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed