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Evaluating Ravulizumab for Children with Neuromyelitis Optica Spectrum Disorder

A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)

Phase2; Phase3 Interventional Alexion Pharmaceuticals, Inc. · NCT05346354

This study is testing if a new medication called ravulizumab can help children with Neuromyelitis Optica Spectrum Disorder feel better and stay safe while using it.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment12 (estimated)
SexAll
SponsorAlexion Pharmaceuticals, Inc. Industry-sponsored
Drugs / interventionsEculizumab, rituximab, methotrexate, cyclophosphamide, ravulizumab
Locations21 sites (Washington D.C., District of Columbia and 20 other locations)
Trial IDNCT05346354 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of ravulizumab, a complement inhibitor, in pediatric patients diagnosed with Neuromyelitis Optica Spectrum Disorder (NMOSD). Participants must be anti-AQP4 antibody positive and have experienced at least one relapse in the past year. The study includes both treatment-naïve patients and those previously treated with eculizumab, ensuring a comprehensive evaluation of the drug's impact on this condition. The study will monitor participants for improvements in their condition and overall safety during the treatment period.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients who are anti-AQP4 antibody positive and have a diagnosis of NMOSD with a recent history of relapse.

Not a fit: Patients who are not anti-AQP4 antibody positive or have not experienced recent relapses may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of NMOSD in children, potentially reducing the frequency and severity of attacks.

How similar studies have performed: Previous studies on complement inhibitors have shown promise in treating NMOSD, suggesting a potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion 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 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 this trial is running

Washington D.C., District of Columbia and 20 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Neuromyelitis Optica Spectrum DisorderRavulizumabALXN1210CNS Autoimmune DisordersNMONMOSD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.