Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
Purpose
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Condition
- Neuromyelitis Optica Spectrum Disorder
Eligibility
- Eligible Ages
- All ages
- Eligible Sex
- All
- Accepts 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.
Study Design
- Phase
- Phase 2/Phase 3
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Ravulizumab |
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. |
|
Recruiting Locations
Research Site
Washington D.C. 4140963, District of Columbia 4138106 20010
Washington D.C. 4140963, District of Columbia 4138106 20010
Research Site
Miami 4164138, Florida 4155751 33136
Miami 4164138, Florida 4155751 33136
Research Site
Boston 4930956, Massachusetts 6254926 02114
Boston 4930956, Massachusetts 6254926 02114
Research Site
Durham 4464368, North Carolina 4482348 27705
Durham 4464368, North Carolina 4482348 27705
Research Site
Philadelphia 4560349, Pennsylvania 6254927 19104
Philadelphia 4560349, Pennsylvania 6254927 19104
More Details
- NCT ID
- NCT05346354
- Status
- Recruiting
- Sponsor
- Alexion Pharmaceuticals, Inc.