An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD

NCT ID: NCT04201262

Last Updated: 2025-10-14

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

58 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-09

Study Completion Date

2024-10-31

Brief Summary

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The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.

Detailed Description

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Conditions

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Neuromyelitis Optica Neuromyelitis Optica Spectrum Disorder

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

External Placebo-Controlled, Open-Label Design
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Ravulizumab

During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed).

After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 3 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.

Group Type EXPERIMENTAL

Ravulizumab

Intervention Type BIOLOGICAL

Participants will receive a weight-based loading dose of ravulizumab via IV infusion on Day 1, followed by weight-based maintenance doses on Day 15, then once every 8 weeks until end of the Long-Term Extension Period.

Interventions

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Ravulizumab

Participants will receive a weight-based loading dose of ravulizumab via IV infusion on Day 1, followed by weight-based maintenance doses on Day 15, then once every 8 weeks until end of the Long-Term Extension Period.

Intervention Type BIOLOGICAL

Other Intervention Names

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ALXN1210 Ultomiris

Eligibility Criteria

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Inclusion Criteria

1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
2. At least 1 attack or relapse in the last 12 months prior to the Screening Period.
3. Expanded Disability Status Scale score ≤7.
4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening.
5. Body weight ≥40 kilograms.
6. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion Criteria

1. History of Neisseria meningitidis infection.
2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer).
3. Previously or currently treated with a complement inhibitor.
4. Use of rituximab or mitoxantrone within 3 months prior to Screening.
5. Use of IV immunoglobulin within 3 weeks prior to Screening.
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

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

Aurora, Colorado, United States

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Fort Collins, Colorado, United States

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

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Miami, Florida, United States

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Boston, Massachusetts, United States

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Rochester, Minnesota, United States

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Jackson, Mississippi, United States

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St Louis, Missouri, United States

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Cincinnati, Ohio, United States

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Houston, Texas, United States

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Camperdown, , Australia

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Fitzroy, , Australia

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Vienna, , Austria

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Burnaby, British Columbia, Canada

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Aarhus, , Denmark

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Strasbourg, , France

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Berlin, , Germany

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Leipzig, , Germany

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München, , Germany

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Cefalù, , Italy

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Gallarate, , Italy

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Napoli, , Italy

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Roma, , Italy

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Rome, , Italy

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Chiba, , Japan

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Fukuoka, , Japan

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Kawagoe-shi, , Japan

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Niigata, , Japan

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Sendai, , Japan

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Sendai, , Japan

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Katowice, , Poland

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Lódz, , Poland

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Goyang-si, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Barcelona, , Spain

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Madrid, , Spain

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Málaga, , Spain

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Oxford, , United Kingdom

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Countries

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United States Australia Austria Canada Denmark France Germany Italy Japan Poland South Korea Spain United Kingdom

References

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Ortiz S, Pittock SJ, Berthele A, Levy M, Nakashima I, Oreja-Guevara C, Allen K, Mashhoon Y, Parks B, Kim HJ. Immediate and sustained terminal complement inhibition with ravulizumab in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. Front Neurol. 2024 Jan 31;15:1332890. doi: 10.3389/fneur.2024.1332890. eCollection 2024.

Reference Type DERIVED
PMID: 38356884 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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CHAMPION-NMO-307

Identifier Type: OTHER

Identifier Source: secondary_id

2019-003352-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALXN1210-NMO-307

Identifier Type: -

Identifier Source: org_study_id

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