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

NCT07221838 | Recruiting Phase 4 FDA Drug

• 2 other identifiers: D9281R00005ALX-MG-502
• Study Type: interventional
• Target: 75
• Locations: 4 countries
• Sites: 12

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.

Conditions

Generalized Myasthenia Gravis; gMG

Keywords

Generalized myasthenia gravis; gMG; Glucocorticoid toxicity; Complement C5 inhibitor; Prednisone reduction; Cortisol monitoring; ravulizumab; corticosteroids; adrenal insufficiency; steroid sparing

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• 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

Study Arms (1)

Oral Corticosteroid Tapering Schedule

EXPERIMENTAL

During the tapering period, participant will follow an Oral Corticosteroid tapering schedule until OCS tapering is complete

Procedure: Oral Corticosteroid Tapering Schedule; Drug: Ravulizumab; Drug: Prednisone/Prednisolone

Interventions

Oral Corticosteroid Tapering Schedule PROCEDURE

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.

Oral Corticosteroid Tapering Schedule

Ravulizumab DRUG

Patients being treated with intravenous ravulizumab as part of their standard medical care.

Oral Corticosteroid Tapering Schedule

Prednisone/Prednisolone DRUG

Participants will continue with or be switched to prednisone/prednisolone as per standard medical care.

Oral Corticosteroid Tapering Schedule

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (12)

Research Site

Chicago, Illinois, 60637, United States

NOT YET RECRUITING

Research Site

Schaumburg, Illinois, 60173, United States

NOT YET RECRUITING

Research Site

Neptune City, New Jersey, 07753, United States

NOT YET RECRUITING

Research Site

Raleigh, North Carolina, 27607, United States

RECRUITING

Research Site

Knoxville, Tennessee, 37920, United States

NOT YET RECRUITING

Research Site

Bochum, 44789, Germany

NOT YET RECRUITING

Research Site

Milan, 20122, Italy

NOT YET RECRUITING

Research Site

Naples, 80131, Italy

NOT YET RECRUITING

Research Site

Rome, 00189, Italy

NOT YET RECRUITING

Research Site

Ibaraki, 305-8576, Japan

NOT YET RECRUITING

Research Site

Kitakyushu-shi, 807-8555, Japan

NOT YET RECRUITING

Research Site

Kumamoto, 860-8556, Japan

NOT YET RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis; Adrenal Insufficiency

Interventions

ravulizumab; Prednisone; Prednisolone

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Adrenal Gland Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Pregnadienetriols

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2025
• First Posted: October 28, 2025
• Study Start: March 4, 2026
• Primary Completion (Estimated): August 27, 2027
• Study Completion (Estimated): August 27, 2027
• Last Updated: April 9, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

US (5); DE (1); IT (3); JP (3)