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

NCT06967480 | Recruiting FDA Drug

• 1 other identifier: D9281R00002
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 23

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.

Conditions

Generalized Myasthenia Gravis; Anti-AChR Antibody Positive

Keywords

Anti-AChR Antibody-Positive; Generalized Myasthenia Gravis; gMG; Ravulizumab

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (24)

• 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

Study Arms (1)

Ravulizumab

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

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

Participants will receive Ravulizumab.

Ravulizumab

Eligibility Criteria

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

Study Population

Patients with anti-AChR antibody positive, generalized, myasthenia gravis elegible to Ravulizumab treatment with a disease duration from onset equal or less than 3 years.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (23)

Clinical Trial Site

Bergamo, Italy

RECRUITING

Clinical Trial Site

Bologna, Italy

NOT YET RECRUITING

Clinical Trial Site

Bologna, Italy

RECRUITING

Clinical Trial Site

Brescia, Italy

RECRUITING

Clinical Trial Site

Florence, Italy

RECRUITING

Clinical Trial Site

Imperia, Italy

NOT YET RECRUITING

Clinical Trial Site

Lecco, Italy

RECRUITING

Clinical Trial Site

Messina, Italy

NOT YET RECRUITING

Clinical Trial Site

Milan, Italy

RECRUITING

Clinical Trial Site

Milan, Italy

NOT YET RECRUITING

Clinical Trial Site

Naples, Italy

NOT YET RECRUITING

Clinical Trial Site

Novara, Italy

NOT YET RECRUITING

Clinical Trial Site

Orbassano, Italy

NOT YET RECRUITING

Clinical Trial Site

Padua, Italy

NOT YET RECRUITING

Clinical Trial Site

Palermo, Italy

NOT YET RECRUITING

Clinical Trial Site

Parma, Italy

RECRUITING

Clinical Trial Site

Pavia, Italy

NOT YET RECRUITING

Clinical Trial Site

Pisa, Italy

NOT YET RECRUITING

Clinical Trial Site

Roma, Italy

NOT YET RECRUITING

Clinical Trial Site

Salerno, Italy

NOT YET RECRUITING

Clinical Trial Site

San Giovanni Rotondo, 71013, Italy

RECRUITING

Clinical Trial Site

Sassari, Italy

RECRUITING

Clinical Trial Site

Torino, Italy

NOT YET RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

ravulizumab

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

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2025
• First Posted: May 13, 2025
• Study Start: July 31, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: February 5, 2026

Record last verified: 2026-02

Data Sharing

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

Locations

IT (23)