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

NCT04201262 | Completed Phase 3 Results FDA Drug

• 3 other identifiers: ALXN1210-NMO-307CHAMPION-NMO-3072019-003352-37
• Study Type: interventional
• Target: 58
• Locations: 12 countries
• Sites: 39

Brief Summary

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

Conditions

Neuromyelitis Optica; Neuromyelitis Optica Spectrum Disorder

Keywords

Neuromyelitis Optica; Neuromyelitis Optica Spectrum Disorder; Devic's Disease; Transverse Myelitis; Optic Neuritis; Relapse; Ravulizumab; Ultomiris; ALXN1210; CNS Autoimmune Disorders; Demyelinating Disorders; NMO; NMOSD

Outcome Measures

Primary Outcomes (1)

• Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period

  An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the independent relapse adjudication committee.

  Baseline up to 2.25 years (end of the Primary Treatment Period)

Secondary Outcomes (9)

• Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period

  Baseline up to 2.25 years (end of the Primary Treatment Period)

• Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period

  Baseline up to 2.25 years (end of the Primary Treatment Period)

• Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period

  Baseline, up to 2.25 years (end of the Primary Treatment Period)

• Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period

  Baseline, up to 2.25 years (end of the Primary Treatment Period)

• Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period

  Baseline up to 2.25 years (end of the Primary Treatment Period)

Study Arms (1)

Ravulizumab

EXPERIMENTAL

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.

Biological: Ravulizumab

Interventions

Ravulizumab 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.

Also known as: ALXN1210, Ultomiris

Ravulizumab

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (41)

Research Site

Aurora, Colorado, 80045, United States

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Fort Collins, Colorado, 80528, United States

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Washington D.C., District of Columbia, 20007, United States

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Miami, Florida, 33136, United States

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Boston, Massachusetts, 02114, United States

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Rochester, Minnesota, 55905, United States

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Jackson, Mississippi, 39216, United States

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St Louis, Missouri, 63110, United States

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Cincinnati, Ohio, 45219, United States

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Houston, Texas, 77030, United States

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Camperdown, 2050, Australia

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Fitzroy, 3065, Australia

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Vienna, 1090, Austria

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Burnaby, British Columbia, V5G 2X6, Canada

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Aarhus, 8200, Denmark

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Strasbourg, 67098, France

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Berlin, 10117, Germany

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Leipzig, 04103, Germany

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München, 81675, Germany

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Cefalù, 90015, Italy

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Gallarate, 21013, Italy

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Napoli, 80131, Italy

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Roma, 00133, Italy

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Rome, 00178, Italy

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Chiba, 260-0877, Japan

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Fukuoka, 812-8582, Japan

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Kawagoe-shi, 350-8550, Japan

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Niigata, 951-8585, Japan

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Sendai, 980-8574, Japan

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Sendai, 983-8512, Japan

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Katowice, 40-571, Poland

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Lódz, 90-324, Poland

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Goyang-si, 10408, South Korea

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Seoul, 02841, South Korea

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Seoul, 03722, South Korea

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Seoul, 06351, South Korea

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Seoul, 143-729, South Korea

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Barcelona, 08036, Spain

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Madrid, 28040, Spain

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Málaga, 29010, Spain

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Oxford, OX3 9DU, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 38356884 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Neuromyelitis Optica; Myelitis, Transverse; Optic Neuritis; Recurrence; Demyelinating Diseases

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis; Central Nervous System Infections; Infections; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Central Nervous System Diseases; Spinal Cord Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: External Placebo-Controlled, Open-Label Design

Study Record Dates

• First Submitted: December 11, 2019
• First Posted: December 17, 2019
• Study Start: December 9, 2019
• Primary Completion: March 15, 2022
• Study Completion: October 31, 2024
• Last Updated: October 14, 2025
• Results First Posted: August 9, 2023

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

ES (3); AU (1); CA (1); DE (3); DK (1); IT (5); JP (6); PL (2); FR (1); US (10); KR (5); GB (1)