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

Alexion Pharmaceuticals

Status: Begins enrollment this month

Conditions: Anti-AChR Antibody Positive; Generalized Myasthenia Gravis

Treatments: Drug: Ravulizumab

Study type: Observational

Funder types: Industry

Identifiers

NCT06967480

D9281R00002

Details and patient eligibility

About

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

Enrollment: 40 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

40 participants in 1 patient group

Ravulizumab

Description:

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

Treatment:

Drug: Ravulizumab