Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)

NCT07557420 | Not Yet Recruiting Phase 3

• 2 other identifiers: D9282C00006ALXN1210-NMO-324
• Study Type: interventional
• Target: 21
• Locations: 0 countries
• Sites: N/A

Brief Summary

The primary objective of this study is to confirm the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of ravulizumab in the treatment of Chinese adults with anti-aquaporin-4 (AQP4) antibody (Ab) + neuromyelitis optica spectrum disorder (NMOSD).

Conditions

NMOSD; Neuromyelitis Optica Spectrum Disorder

Keywords

Ravulizumab; NMOSD; Neuromyelitis optica spectrum disorder

Outcome Measures

Primary Outcomes (1)

• Adjudicated On-Trial Annualized Relapse Rate (ARR)

  Baseline up to Week 50

Secondary Outcomes (9)

• Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score

  Baseline up to Week 50

• Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score

  Baseline up to Week 50

• Change From Baseline in European Quality of Life Health 5-item Questionnaire (EQ-5D) Index Score

  Baseline, Week 50

• Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score

  Baseline, Week 50

• Number of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interests (AESIs)

  Baseline up to Week 50

Study Arms (1)

Ravulizumab

EXPERIMENTAL

Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by a weight-based maintenance treatment dose on Day 15 and every 8 weeks (q8w) thereafter for up to 50 weeks.

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

Participants will receive ravulizumab via intravenous (IV) infusion.

Ravulizumab

Eligibility Criteria

• Age: 18 Years - 130 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

Key Inclusion (essential) * Diagnosis: NMOSD per 2015 international consensus criteria, and anti AQP4 antibody positive at Screening. * Disease activity: ≥1 attack/relapse in the past 12 months. * Disability: EDSS ≤7. * Background therapy: If on IST and/or oral corticosteroids, participant should be on a stable maintenance regimen prior to Screening and plan to remain stable during the study unless relapse occurs. (Detailed agent specific duration/dose rules to be confirmed at screening.) * Body weight: ≥40 kg. * Vaccinated against meningococcal infections from serogroups A, C, W, Y (and B where available) within the 3 years prior to study intervention administration on Day 1. Key Exclusion (essential) * Pregnancy/lactation: Pregnant, breastfeeding, or intending to conceive during the study. * Infection risk: History of meningococcal disease or unresolved meningococcal disease, active systemic infection within 14 days, or fever ≥38°C within 7 days before Day 1. * Hypersensitivity: To murine proteins or ravulizumab excipients. * Serious comorbidities: Any condition that in the Investigator's judgment adds risk or interferes with participation/assessment. * Viral infections: Known HIV, active HBV, or active HCV. * Prior/concomitant immunomodulatory treatments: * B cell-depleting therapy (e.g., rituximab, inebilizumab) within 3 months before Screening. * Mitoxantrone or satralizumab within 3 months before Screening. * IVIg within 3 weeks before Screening. * Any prior or current complement inhibitor. Note: Other protocol-defined criteria may apply and should be verified during full eligibility review.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

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

Study Design

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

Study Record Dates

• First Submitted: April 23, 2026
• First Posted: April 29, 2026
• Study Start (Estimated): August 31, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR