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

Alexion Pharmaceuticals, Inc.40 enrolled

Overview

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

Generalized Myasthenia Gravis Anti-AChR Antibody Positive

Interventions

RavulizumabDRUG

Participants will receive Ravulizumab.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (23)

Clinical Trial Site

Bergamo, Italy

RECRUITING

Outcomes

Primary Outcomes

Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 50

Time frame: Week 50

Change From Baseline in MG-ADL Total Score at Week 50

Time frame: Baseline, Week 50

Secondary Outcomes

MG-ADL Total Score at Weeks 2, 10, and 26

Time frame: Weeks 2, 10, and 26

Change From Baseline in MG-ADL Total Score at Weeks 2, 10 and 26

Time frame: 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

Time frame: Baseline, Weeks 2, 10, 26, and 50

Quantitative Myasthenia Gravis (QMG) Total Score at Weeks 2, 10, 26, and 50

Time frame: Weeks 2, 10, 26, and 50

Change From Baseline in QMG Total Score at Weeks 2, 10, 26 and 50

Time frame: 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

Time frame: Baseline, Weeks 2, 10, 26, and 50

Myasthenia Gravis Composite (MGC) Total Score at Weeks 2, 10, 26, and 50

Time frame: Weeks 2, 10, 26, and 50

Change From Baseline in MGC Total Score at Weeks 2,10, 26 and 50

Time frame: Baseline, Weeks 2, 10, 26 and 50

Central Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

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

Data from ClinicalTrials.gov

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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

...and 13 more locations

Percentage of Participants With >=3- Point Reduction From Baseline in MGC Total Score at Weeks 2, 10, 26 and 50

Time frame: 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

Time frame: Weeks 10, 26, and 50

Change From Baseline in MG-QoL15r Total Score at Weeks 10, 26 and 50

Time frame: Baseline, Weeks 10, 26 and 50

Hospital Anxiety and Depression Scale (HADS) Total Score at Weeks 10, 26, and 50

Time frame: Weeks 10, 26, and 50

Change From Baseline in HADS Total Score at Weeks 10, 26 and 50

Time frame: Baseline, Weeks 10, 26 and 50

Insomnia Severity Index Scale (ISI) Total Score at Weeks 10, 26, and 50

Time frame: Weeks 10, 26, and 50

Change From Baseline in ISI Total Score at Weeks 10, 26 and 50

Time frame: Baseline, Weeks 10, 26 and 50

Pain Visual Analogue Scale (Pain VAS) Total Score at Weeks 2, 10, 26, and 50

Time frame: Weeks 2, 10, 26, and 50

Change From Baseline in Pain VAS Total Score at Weeks 2, 10, 26 and 50

Time frame: Baseline, Weeks 2, 10, 26 and 50

Percentage of Participants With Mean Dose Reduction From Baseline of Corticosteroids and Immunosuppressants (ISTs) Treatment at Weeks 2, 10, 26, and 50

Time frame: Baseline, Weeks 2, 10, 26, and 50

Percentage of Participants in Different ISTs Status at Baseline, and at Weeks 2, 10, 26, and 50

Time frame: Baseline, Weeks 2, 10, 26, and 50

Number of Clinical Deterioration Events

Time frame: Baseline, Weeks 2, 10, 26, and 50

Percentage of Participants With Minimal Symptom Expression at Weeks 2, 10, 26, and 50

Time frame: Weeks 2, 10, 26, and 50

Institute For Medical Technology Assessment (iMTA) Productivity Cost Questionnaire (iPCQ) Score at Weeks 10, 26, and 50

Time frame: Weeks 10, 26, and 50

Change From Baseline in iMTA iPCQ Score at Weeks 10, 26 and 50

Time frame: Baseline, Weeks 10, 26 and 50

Biomarkers analyses (complement system proteins, interleukins and miRNA serum levels)

Time frame: Baseline, Week 10, 26, and 50