Ravulizumab: 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.
Study summary
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
* Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
* Expanded Disability Status Scale (EDSS) score ≤ 7.
* Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1.
* Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine \[AZA\], mycophenolate mofetil \[MMF\], methotrexate \[MTX\], tacrolimus \[TAC\], cyclosporin \[CsA\], or cyclophosphamide \[CYC\]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
* To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection.
* Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group.
Exclusion Criteria:
* Use of rituximab within 3 months prior to screening.
* Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
* Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
* Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
* Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
Primary outcome measure(s)
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
Trial sites (16)
Facility
City
Region
Status
Research Site
Washington D.C.
District of Columbia
Research Site
Miami
Florida
Research Site
Boston
Massachusetts
Research Site
Durham
North Carolina
Research Site
Philadelphia
Pennsylvania
Research Site
Edmonton
Alberta
Research Site
Toronto
Ontario
Research Site
Montreal
Quebec
Research Site
Le Kremlin-Bicêtre
France
Research Site
Marseille
France
Research Site
Chieti
Italy
Research Site
Gallarate
Italy
Research Site
Roma
Italy
Research Site
Yokohama
Japan
Research Site
Goyang-si
South Korea
Research Site
Esplugues de Llobregat
Spain
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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