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The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab Intravenous (IV) Infusion | Experimental | All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Ravulizumab will be administered by intravenous (IV) infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Serum Concentration of Ravulizumab | Day 1 predose through Week 18 predose | |
| Serum Free C5 Concentration | Day 1 predose through Week 18 predose |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18 | Baseline, Up to Week 18 | |
| Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18 | Baseline, Up to Week 18 |
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Inclusion Criteria:
Exclusion Criteria:
Medical Conditions
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Los Angeles | California | 90078 | United States | ||
| Research 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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| Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18 | Baseline, Up to Week 18 |
| Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18 | Week 10, Up to Week 18 |
| Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18 | Participants ≥8 years of age will be evaluated. | Baseline, Up to Week 18 |
| Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18 | Participants <8 years of age will be evaluated. | Baseline, Up to Week 18 |
| Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18 | Baseline through Week 18 |
| Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18 | Baseline through Week 18 |
| Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline | Stable is defined as a ±5-point change from Baseline. | Baseline through Week 18 |
| Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline | Stable is defined as a ±3-point change from baseline. | Baseline through Week 18 |
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events | Baseline up to Week 126 (8 weeks after last dose of study drug) |
| Number of Participants With Anti-Drug Antibody (ADA) at Week 18 | Baseline through Week 18 |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| Research Site | Chapel Hill | North Carolina | 27514 | United States |
| Research Site | Philadelphia | Pennsylvania | 19104 | United States |
| Research Site | Milan | 20133 | Italy |
| Research Site | Roma | 00165 | Italy |
| Research Site | Torino | 10126 | Italy |
| Research Site | Itabashi-ku | 173-0003 | Japan |
| Research Site | Belgrade | 11000 | Serbia |
| Research Site | Belgrade | 11070 | Serbia |
| Research Site | Bern | 3010 | Switzerland |
| 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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