A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab

Not Applicable

Not yet recruiting

• Conditions: Generalized Myasthenia Gravis; gMG
• Interventions: Procedure: Oral Corticosteroid Tapering Schedule; Drug: Ravulizumab; Drug: Prednisone/Prednisolone

• Registration Number: NCT07221838
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-10
• Study First Submitted: 2025-10-03
• Study First Submitted QC: 2025-10-24
• Last Update Submitted: 2025-10-24
• Study First Posted: 2025-10-28
• Last Update Posted: 2025-10-28
• Study Start: 2025-12-31
• Primary Completion: 2027-06-30
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 75

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Oral Corticosteroid Tapering Schedule	Oral Corticosteroid Tapering Schedule	During the tapering period, participant will follow an Oral Corticosteroid tapering schedule until OCS tapering is complete
Oral Corticosteroid Tapering Schedule	Ravulizumab	During the tapering period, participant will follow an Oral Corticosteroid tapering schedule until OCS tapering is complete
Oral Corticosteroid Tapering Schedule	Prednisone/Prednisolone	During the tapering period, participant will follow an Oral Corticosteroid tapering schedule until OCS tapering is complete

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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