The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
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.
Research Site
Washington D.C., District of Columbia, United States
RECRUITINGChange From Baseline in the Annualized Relapse Rate at Week 50
Time frame: Baseline, Week 50
Time to First Adjudicated On-trial Relapse through Week 50
Time frame: Baseline through Week 50
Change From Baseline in Expanded Disability Status Scale Score At Week 50
The score ranges are 0 to 10, higher score indicates worse outcome.
Time frame: Baseline, Week 50
Change From Baseline in Hauser Ambulation Index at Week 50
The score ranges are 0 to 9, higher score indicates worse outcome.
Time frame: Baseline, Week 50
Change From Baseline in Visual Acuity at Week 50
Time frame: Baseline, Week 50
Change From Baseline in Confrontational Visual Fields at Week 50
Time frame: Baseline, Week 50
Change From Baseline in Color Vision at Week 50
Time frame: Baseline, Week 50
Serum Ravulizumab Concentration
Time frame: 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
Time frame: Baseline through Week 50
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Research Site
Miami, Florida, United States
RECRUITINGResearch Site
Boston, Massachusetts, United States
RECRUITINGResearch Site
St Louis, Missouri, United States
WITHDRAWNResearch Site
Durham, North Carolina, United States
RECRUITINGResearch Site
Philadelphia, Pennsylvania, United States
RECRUITINGResearch Site
Edmonton, Alberta, Canada
RECRUITINGResearch Site
Toronto, Ontario, Canada
RECRUITINGResearch Site
Montreal, Quebec, Canada
RECRUITINGResearch Site
Le Kremlin-Bicêtre, France
RECRUITING...and 11 more locations