Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

Running, not enrolling · Phase 2/Phase 3

Conditions studied: Neuromyelitis Optica Spectrum Disorder

In brief

The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).

Key facts

Study ID

NCT05346354

Run by

Alexion Pharmaceuticals, Inc.

People needed

12

Starts

2022-06-23

Expected to finish

2029-03-30

Last updated by the study team

2026-06-24

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Research Site — Washington D.C., District of Columbia, United States
• Research Site — Miami, Florida, United States
• Research Site — Boston, Massachusetts, United States
• Research Site — Durham, North Carolina, United States
• Research Site — Philadelphia, Pennsylvania, United States
• Research Site — Edmonton, Alberta, Canada
• Research Site — Toronto, Ontario, Canada
• Research Site — Montreal, Quebec, Canada
• 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

Full record on ClinicalTrials.gov

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