NCT05346354

Brief 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).

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_2

Timeline
36mo left

Started Jun 2022

Longer than P75 for phase_2

Geographic Reach
8 countries

21 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress57%
Jun 2022Apr 2029

First Submitted

Initial submission to the registry

April 20, 2022

Completed
6 days until next milestone

First Posted

Study publicly available on registry

April 26, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

June 23, 2022

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2026

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 2, 2029

Expected
Last Updated

November 12, 2025

Status Verified

November 1, 2025

Enrollment Period

3.8 years

First QC Date

April 20, 2022

Last Update Submit

November 11, 2025

Conditions

Neuromyelitis Optica Spectrum Disorder

Keywords

Neuromyelitis Optica Spectrum DisorderRavulizumabALXN1210CNS Autoimmune DisordersNMONMOSD

Outcome Measures

Primary Outcomes (2)

  • Change From Baseline in the Annualized Relapse Rate at Week 50

    Baseline, Week 50

  • Time to First Adjudicated On-trial Relapse through Week 50

    Baseline through Week 50

Secondary Outcomes (7)

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

    Baseline, Week 50

  • Change From Baseline in Hauser Ambulation Index at Week 50

    Baseline, Week 50

  • Change From Baseline in Visual Acuity at Week 50

    Baseline, Week 50

  • Change From Baseline in Confrontational Visual Fields at Week 50

    Baseline, Week 50

  • Change From Baseline in Color Vision at Week 50

    Baseline, Week 50

  • +2 more secondary outcomes

Study Arms (1)

Ravulizumab

EXPERIMENTAL

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.

Drug: Ravulizumab

Interventions

RavulizumabDRUG

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.

Also known as: ALXN1210, Ultomiris
Ravulizumab

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (21)

Research Site

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Research Site

Miami, Florida, 33136, United States

RECRUITING

Research Site

Boston, Massachusetts, 02114, United States

RECRUITING

Research Site

St Louis, Missouri, 63110, United States

WITHDRAWN

Research Site

Durham, North Carolina, 27705, United States

RECRUITING

Research Site

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Research Site

Edmonton, Alberta, T6G 1C9, Canada

RECRUITING

Research Site

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Research Site

Montreal, Quebec, H3T1C5, Canada

RECRUITING

Research Site

Le Kremlin-Bicêtre, 94270, France

RECRUITING

Research Site

Marseille, 13005, France

RECRUITING

Research Site

Montpellier, 34295, France

RECRUITING

Research Site

Bochum, 44791, Germany

WITHDRAWN

Research Site

Giessen, 35392, Germany

WITHDRAWN

Research Site

Catania, 95123, Italy

WITHDRAWN

Research Site

Chieti, 66013, Italy

RECRUITING

Research Site

Gallarate, 21013, Italy

RECRUITING

Research Site

Roma, 00165, Italy

RECRUITING

Research Site

Yokohama, 232-0024, Japan

RECRUITING

Research Site

Goyang-si, 10408, South Korea

RECRUITING

Research Site

Esplugues de Llobregat, 8950, Spain

RECRUITING

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Myelitis, TransverseDemyelinating Autoimmune Diseases, CNSAutoimmune Diseases of the Nervous SystemNervous System DiseasesOptic NeuritisOptic Nerve DiseasesCranial Nerve DiseasesDemyelinating DiseasesEye DiseasesAutoimmune DiseasesImmune System Diseases

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356clinicaltrials@alexion.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 20, 2022

First Posted

April 26, 2022

Study Start

June 23, 2022

Primary Completion

March 31, 2026

Study Completion (Estimated)

April 2, 2029

Last Updated

November 12, 2025

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will share

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Locations

FR(3)JP(1)CA(3)IT(4)DE(2)US(6)ES(1)KR(1)