Anti-AChR Antibody Positive, Generalized Myasthenia Gravis
Conditions
Keywords
Anti-AChR Antibody-Positive, Generalized Myasthenia Gravis, gMG, Ravulizumab
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.
Interventions
DRUGRavulizumab
Participants will receive Ravulizumab.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| 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 |
| Biomarkers analyses (complement system proteins, interleukins and miRNA serum levels) | Baseline, Week 10, 26, and 50 |
| MG-ADL Total Score at Weeks 2, 10, and 26 | Weeks 2, 10, and 26 |
Countries
Italy
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed