Neuromyelitis Optica Spectrum Disorder, NMOSD
Conditions
Keywords
Ravulizumab, NMOSD, Neuromyelitis optica spectrum disorder
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).
Interventions
Participants will receive ravulizumab via intravenous (IV) infusion.
Sponsors
Study design
Eligibility
Inclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adjudicated On-Trial Annualized Relapse Rate (ARR) | Baseline up to Week 50 |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| Serum Ravulizumab Concentration | Day 1 to Day 351 |
| Change From Baseline in Serum Free Complement Component 5 (C5) Concentration | Baseline Up to Day 351 |
| Number of Participants With Anti-Drug Antibodies (ADAs) | Day 1 up to Day 351 |
| Number of Participants With Neutralizing Antibodies (NAb) | Day 1 up to Day 351 |
Countries
China