Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis

Not yet recruiting

• Conditions: Generalized Myasthenia Gravis; Anti-AChR Antibody Positive
• Interventions: Drug: Ravulizumab

• Registration Number: NCT06967480
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-05
• Study First Submitted: 2025-05-05
• Study First Submitted QC: 2025-05-05
• Last Update Submitted: 2025-05-05
• Study First Posted: 2025-05-13
• Last Update Posted: 2025-05-13
• Study Start: 2025-06-13
• Primary Completion: 2027-05-31
• Study Completion: 2027-05-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Diagnosis of MG confirmed by:

  • History of a positive serologic test for anti-AChR antibodies, and

  • One of the following:

    • History of abnormal neuromuscular transmission test demonstrated by singlefibre electromyography or repetitive nerve stimulation
    • History of positive anticholinesterase test (e.g., edrophonium chloride test); or
    • Patient demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician

• Disease duration from MG onset ≤ 3 years before T-4 (Enrolment);

• MGFA class IIb to IV;

• Patient eligible for Ravulizumab treatment based on AIFA reimbursement criteria

• Vaccination cycle for Neisseria meningitidis completed at least two weeks before Ravulizumab initiation or antibiotics chemoprophylaxis according to the SmPC.

Exclusion Criteria

• Patient unable to understand and sign the informed consent
• Hypersensitivity to the active substance or to any of the excipients of the study product
• Patient for whom the study product is contraindicated according to SmPC
• Previous treatment with C5 inhibitors
• Last rituximab infusion performed < 6 months before T-4 (Enrolment)
• Last infusion with FcRn blockers performed < 3 months before T-4 (Enrollment)
• Pregnant or lactating or planning a pregnancy during the study
• Patient who plan to relocate during the study
• Patient who are unsure of following the visit schedule
• Patient unable to complete questionnaires
• Previous or current participation to other interventional studies

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Ravulizumab	Ravulizumab	Participants will receive Ravulizumab until Week 50 ± 14 days after baseline or until discontinuation.

Primary Outcome Measures

Name	Time	Method
Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 50	Week 50
Change From Baseline in MG-ADL Total Score at Week 50	Baseline, Week 50

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With Mean Dose Reduction From Baseline of Corticosteroids and Immunosuppressants (ISTs) Treatment at Weeks 2, 10, 26, and 50	Baseline, Weeks 2, 10, 26, and 50
Percentage of Participants in Different ISTs Status at Baseline, and at Weeks 2, 10, 26, and 50	Baseline, Weeks 2, 10, 26, and 50
Number of Clinical Deterioration Events	Baseline, Weeks 2, 10, 26, and 50
Percentage of Participants With Minimal Symptom Expression at Weeks 2, 10, 26, and 50	Weeks 2, 10, 26, and 50
Institute For Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ) Score at Weeks 10, 26, and 50	Weeks 10, 26, and 50
Change From Baseline in iMTA iPCQ Score at Weeks 10, 26 and 50	Baseline, Weeks 10, 26 and 50
MG-ADL Total Score at Weeks 2, 10, and 26	Weeks 2, 10, and 26
Change From Baseline in MG-ADL Total Score at Weeks 2, 10 and 26	Baseline, Weeks 2, 10 and 26
Percentage of Participants With >=2- Point Reduction From Baseline in MG-ADL Total Score at Weeks 2, 10, 26, and 50	Baseline, Weeks 2, 10, 26, and 50
Quantitative Myasthenia Gravis (QMG) Total Score at Weeks 2, 10, 26, and 50	Weeks 2, 10, 26, and 50
Change From Baseline in QMG Total Score at Weeks 2, 10, 26 and 50	Baseline, Weeks 2, 10, 26 and 50
Percentage of Participants With >=3- Point Reduction From Baseline in QMG Total Score at Weeks 2, 10, 26, and 50	Baseline, Weeks 2, 10, 26, and 50
Myasthenia Gravis Composite (MGC) Total Score at Weeks 2, 10, 26, and 50	Weeks 2, 10, 26, and 50
Change From Baseline in MGC Total Score at Weeks 2,10, 26 and 50	Baseline, Weeks 2, 10, 26 and 50
Percentage of Participants With >=3- Point Reduction From Baseline in MGC Total Score at Weeks 2, 10, 26 and 50	Baseline, Weeks 2, 10, 26, and 50
Myasthenia Gravis Quality of Life 15-Item Scale - Revised (MG-QoL15r) Total Score at Weeks 10, 26, and 50	Weeks 10, 26, and 50
Change From Baseline in MG-QoL15r Total Score at Weeks 10, 26 and 50	Baseline, Weeks 10, 26 and 50
Hospital Anxiety and Depression Scale (HADS) Total Score at Weeks 10, 26, and 50	Weeks 10, 26, and 50
Change From Baseline in HADS Total Score at Weeks 10, 26 and 50	Baseline, Weeks 10, 26 and 50
Insomnia Severity Index Scale (ISI) Total Score at Weeks 10, 26, and 50	Weeks 10, 26, and 50
Change From Baseline in ISI Total Score at Weeks 10, 26 and 50	Baseline, Weeks 10, 26 and 50
Pain Visual Analogue Scale (Pain VAS) Total Score at Weeks 2, 10, 26, and 50	Weeks 2, 10, 26, and 50
Change From Baseline in Pain VAS Total Score at Weeks 2, 10, 26 and 50	Baseline, Weeks 2, 10, 26 and 50
Biomarkers analyses (complement system proteins, interleukins and miRNA serum levels)	Baseline, Week 10, 26, and 50

Trial Locations

Locations (1)

Clinical Trial Site; 🇮🇹 Torino, Italy