Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Ravulizumab.
- Who it may be relevant to
- Registry conditions: NMOSD, Neuromyelitis Optica Spectrum Disorder. Basic parameters: 18 years — 130 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3b, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)
Overview
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
- Drug Ravulizumab
Participants will receive ravulizumab via intravenous (IV) infusion.
Primary outcome measures
- Adjudicated On-Trial Annualized Relapse Rate (ARR) [Time frame: Baseline up to Week 50]
Secondary outcome measures (9)
- Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score [Time frame: Baseline up to Week 50]
- Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score [Time frame: Baseline up to Week 50]
- Change From Baseline in European Quality of Life Health 5-item Questionnaire (EQ-5D) Index Score [Time frame: Baseline, Week 50]
- Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score [Time frame: 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) [Time frame: Baseline up to Week 50]
- Serum Ravulizumab Concentration [Time frame: Day 1 to Day 351]
- Change From Baseline in Serum Free Complement Component 5 (C5) Concentration [Time frame: Baseline Up to Day 351]
- Number of Participants With Anti-Drug Antibodies (ADAs) [Time frame: Day 1 up to Day 351]
- Number of Participants With Neutralizing Antibodies (NAb) [Time frame: Day 1 up to Day 351]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 5 centers
- Research Site — Chengdu
- Research Site — Dongguan
- Research Site — Shanghai
- Research Site — Wenzhou
- Research Site — Wuhan
Identifiers
NCT: NCT07557420 · D9282C00006 · ALXN1210-NMO-324