Overview

Phase 2/3 Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

Status:
Not yet recruiting
Trial end date:
2028-03-30
Target enrollment:
0
Participant gender:
All
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).
Phase:
Phase 2/Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alexion Pharmaceuticals
Treatments:
Ravulizumab
Criteria
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 6 months prior to Day 1.

- 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.