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NCT Number: NCT05346354

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

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Neuromyelitis Optica Spectrum Disorder Autoimmune Diseases Autoimmune Diseases of the Nervous System Cranial Nerve Diseases Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Eye Diseases Immune System Diseases Myelitis, Transverse Nervous System Diseases Neuromyelitis Optica Optic Nerve Diseases Optic Neuritis

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Research Site, Edmonton, Alberta, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Ravulizumab

Drug

Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight.

During the Extension Period, participants will continue to receive weight-based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight.

Other names: ALXN1210, Ultomiris

Primary outcomes

  1. Change From Baseline in the Annualized Relapse Rate at Week 50

    Time frame: Baseline, Week 50

  2. Time to First Adjudicated On-trial Relapse through Week 50

    Time frame: Baseline through Week 50

Secondary outcomes

  1. Change From Baseline in Expanded Disability Status Scale Score At Week 50

    Time frame: Baseline, Week 50

    The score ranges are 0 to 10, higher score indicates worse outcome.

  2. Change From Baseline in Hauser Ambulation Index at Week 50

    Time frame: Baseline, Week 50

    The score ranges are 0 to 9, higher score indicates worse outcome.

  3. Change From Baseline in Visual Acuity at Week 50

    Time frame: Baseline, Week 50

  4. Change From Baseline in Confrontational Visual Fields at Week 50

    Time frame: Baseline, Week 50

  5. Change From Baseline in Color Vision at Week 50

    Time frame: Baseline, Week 50

  6. Serum Ravulizumab Concentration

    Time frame: Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50

  7. Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50

    Time frame: Baseline through Week 50

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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)

Important dates

Study start
2022
Primary completion
2026
Study completion
2029
First posted
Apr 26, 2022
Registry last updated
Jun 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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