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The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab | Experimental | During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment. During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight. During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Annualized Relapse Rate at Week 50 | Baseline, Week 50 | |
| Time to First Adjudicated On-trial Relapse through Week 50 | Baseline through Week 50 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Expanded Disability Status Scale Score At Week 50 | The score ranges are 0 to 10, higher score indicates worse outcome. | Baseline, Week 50 |
| Change From Baseline in Hauser Ambulation Index at Week 50 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Washington D.C. | District of Columbia | 20010 | 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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The score ranges are 0 to 9, higher score indicates worse outcome.
| Baseline, Week 50 |
| Change From Baseline in Visual Acuity at Week 50 | Baseline, Week 50 |
| Change From Baseline in Confrontational Visual Fields at Week 50 | Baseline, Week 50 |
| Change From Baseline in Color Vision at Week 50 | Baseline, Week 50 |
| Serum Ravulizumab Concentration | Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50 |
| Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50 | Baseline through Week 50 |
| Miami |
| Florida |
| 33136 |
| United States |
| Research Site | Boston | Massachusetts | 02114 | United States |
| Research Site | Durham | North Carolina | 27705 | United States |
| Research Site | Philadelphia | Pennsylvania | 19104 | United States |
| Research Site | Edmonton | Alberta | T6G 1C9 | Canada |
| Research Site | Toronto | Ontario | M5G 1X8 | Canada |
| Research Site | Montreal | Quebec | H3T1C5 | Canada |
| Research Site | Le Kremlin-BicĂȘtre | 94270 | France |
| Research Site | Marseille | 13005 | France |
| Research Site | Chieti | 66013 | Italy |
| Research Site | Gallarate | 21013 | Italy |
| Research Site | Roma | 00165 | Italy |
| Research Site | Yokohama | 232-0024 | Japan |
| Research Site | Goyang-si | 10408 | South Korea |
| Research Site | Esplugues de Llobregat | 8950 | Spain |
| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000629409 | ravulizumab |
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