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

NCT ID: NCT05346354

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-23

Study Completion Date

2029-04-02

Brief Summary

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

Detailed Description

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Conditions

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Neuromyelitis Optica Spectrum Disorder

Keywords

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Neuromyelitis Optica Spectrum Disorder Ravulizumab ALXN1210 CNS Autoimmune Disorders NMO NMOSD

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Ravulizumab

Intervention Type 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.

Interventions

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Ravulizumab

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.

Intervention Type DRUG

Other Intervention Names

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ALXN1210 Ultomiris

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

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

Site Status RECRUITING

Research Site

Miami, Florida, United States

Site Status RECRUITING

Research Site

Boston, Massachusetts, United States

Site Status RECRUITING

Research Site

St Louis, Missouri, United States

Site Status WITHDRAWN

Research Site

Durham, North Carolina, United States

Site Status RECRUITING

Research Site

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Research Site

Edmonton, Alberta, Canada

Site Status RECRUITING

Research Site

Toronto, Ontario, Canada

Site Status RECRUITING

Research Site

Montreal, Quebec, Canada

Site Status RECRUITING

Research Site

Le Kremlin-Bicêtre, , France

Site Status RECRUITING

Research Site

Marseille, , France

Site Status RECRUITING

Research Site

Montpellier, , France

Site Status RECRUITING

Research Site

Bochum, , Germany

Site Status WITHDRAWN

Research Site

Giessen, , Germany

Site Status WITHDRAWN

Research Site

Catania, , Italy

Site Status WITHDRAWN

Research Site

Chieti, , Italy

Site Status RECRUITING

Research Site

Gallarate, , Italy

Site Status RECRUITING

Research Site

Roma, , Italy

Site Status RECRUITING

Research Site

Yokohama, , Japan

Site Status RECRUITING

Research Site

Goyang-si, , South Korea

Site Status RECRUITING

Research Site

Esplugues de Llobregat, , Spain

Site Status RECRUITING

Countries

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United States Canada France Germany Italy Japan South Korea Spain

Central Contacts

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Alexion Pharmaceuticals, Inc. (Sponsor)

Role: CONTACT

Phone: 1-855-752-2356

Email: [email protected]

Other Identifiers

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ALXN1210-NMO-317

Identifier Type: -

Identifier Source: org_study_id