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The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab | Participants will receive Ravulizumab until Week 50 ± 14 days after baseline or until discontinuation. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Participants will receive Ravulizumab. |
|
| Measure | Description | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
Diagnosis of MG confirmed by:
History of a positive serologic test for anti-AChR antibodies, and
One of the following:
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:
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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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. (Sponsor) | Contact | 1-855-752-2356 | clinicaltrials@alexion.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Recruiting | Bergamo | Italy | |||
| Clinical Trial Site |
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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Blood
| 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 |
| Not yet recruiting |
| Bologna |
| Italy |
| Clinical Trial Site | Recruiting | Bologna | Italy |
| Clinical Trial Site | Recruiting | Brescia | Italy |
| Clinical Trial Site | Recruiting | Florence | Italy |
| Clinical Trial Site | Not yet recruiting | Imperia | Italy |
| Clinical Trial Site | Recruiting | Lecco | Italy |
| Clinical Trial Site | Not yet recruiting | Messina | Italy |
| Clinical Trial Site | Recruiting | Milan | Italy |
| Clinical Trial Site | Not yet recruiting | Milan | Italy |
| Clinical Trial Site | Not yet recruiting | Naples | Italy |
| Clinical Trial Site | Not yet recruiting | Novara | Italy |
| Clinical Trial Site | Not yet recruiting | Orbassano | Italy |
| Clinical Trial Site | Not yet recruiting | Padova | Italy |
| Clinical Trial Site | Not yet recruiting | Palermo | Italy |
| Clinical Trial Site | Recruiting | Parma | Italy |
| Clinical Trial Site | Not yet recruiting | Pavia | Italy |
| Clinical Trial Site | Not yet recruiting | Pisa | Italy |
| Clinical Trial Site | Not yet recruiting | Roma | Italy |
| Clinical Trial Site | Not yet recruiting | Salerno | Italy |
| Clinical Trial Site | Recruiting | San Giovanni Rotondo | 71013 | Italy |
| Clinical Trial Site | Recruiting | Sassari | Italy |
| Clinical Trial Site | Not yet recruiting | Torino | Italy |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000629409 | ravulizumab |
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