Back to search results

Study of Ravulizumab for Children with NMOSD

This study is looking at the safety and effectiveness of a medicine called ravulizumab in children with Neuromyelitis Optica Spectrum Disorder (NMOSD), a rare, lifelong autoimmune disease of the central nervous system. To join, children must have NMOSD, test positive for a specific antibody (anti-AQP4 Ab), and have experienced at least one attack or relapse in the past year. They also need to have an EDSS score (a measure of disability) of 7 or less. Participants will receive ravulizumab through an IV, starting with a loading dose, then maintenance doses every 4 or 8 weeks depending on their weight. The study aims to see if ravulizumab can reduce the number of relapses over 50 weeks and how long it takes for the first relapse to occur. The study plans to enroll 12 participants and its current status is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is not specified if it's randomized or blinded, and the phase is not specified. It plans to enroll 12 participants.
What's involved
Participants will receive ravulizumab through an IV on Day 1, Day 15, and then every 4 or 8 weeks depending on their weight.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoints are measured at Week 50, indicating follow-up for at least that duration.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05346354

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

Active, Not Recruiting
PHASE2All AgesInterventionalTreatment
Alexion Pharmaceuticals, Inc.
~12 participants
Updated 2026-06-24 on ClinicalTrials.gov
What's tested:Ravulizumab

At a glance

Recruiting sites
0 of 16 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change From Baseline in the Annualized Relapse Rate at Week 50
Measured over Baseline, Week 50
+1 more outcome measured

Conditions

Neuromyelitis Optica Spectrum Disorder

Explore related trials

Neuromyelitis OpticaAQP4 Gene

Where it's being run

16 sites across 13 states
Italy3
France2
District of Columbia1
Florida1
Massachusetts1
North Carolina1
Pennsylvania1
Alberta1

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Change From Baseline in the Annualized Relapse Rate at Week 50Baseline, Week 50
  • Time to First Adjudicated On-trial Relapse through Week 50Baseline through Week 50