Phase 3
Completed N=105
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
Source: ClinicalTrials.gov NCT04201262 ↗Enrolled (actual)
105
Serious AEs
26.7%
Results posted
Aug 2023
Primary outcomePrimary: Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period — 0; 20 Participants — p=< 0.0001
◆ Published Evidence
Emerging
9citations · ~5 / year
Immediate and sustained terminal complement inhibition with ravulizumab in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.
Summary
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
Linked Publications (2)
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Immediate and sustained terminal complement inhibition with ravulizumab in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.
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Safety and efficacy of ravulizumab in patients with NMOSD previously treated with rituximab: A post hoc analysis of the CHAMPION-NMOSD trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period |
0; 20 | < 0.0001 sig |
| SECONDARY Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period |
0.000; 0.350 | — |
| SECONDARY Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period |
4; 4; 52; 32; 2; 11 | 0.0122 sig |
| SECONDARY Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period |
0.005; -0.043 | — |
| SECONDARY Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period |
2.6; 0.6 | — |
| SECONDARY Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period |
52; 36; 6; 11 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Study Drug Discontinuation in the Primary Treatment Period |
53; 45; 8; 26; 1; 2 | — |
| SECONDARY Serum Ravulizumab Concentration |
760.3; 1836.4 | — |
| SECONDARY Change From Baseline in Serum Free C5 Concentration at Week 26 |
-119.02; -119.32 | — |
| SECONDARY Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period |
5; 53; 1; 54; 0; 52 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT04201262) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.