Phase 3
N=105
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD
Neuromyelitis Optica · Neuromyelitis Optica Spectrum Disorder
Bottom Line
View on ClinicalTrials.gov: NCT04201262 ↗Enrolled (actual)
105
Serious AEs
26.7%
Results posted
Aug 2023
Primary outcome: Primary: Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period — 0; 20 Participants — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ravulizumab (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Mar 2022
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 | — |
Summary
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
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). 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.