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A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD

A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
CompletedPhase 3NMOSDNCT042012622019-003352-37

What it found

Met its main goal

The main measure showed a statistically significant benefit.

MeasureEstimate95% CIp
Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment PeriodHazard Ratio (HR) 0.0140.000–0.103< 0.0001

Statistical analysis as posted on ClinicalTrials.gov.

What was tested

Treatment tested

Results by group

Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period

Baseline up to 2.25 years (end of the Primary Treatment Period)

GroupValueIntervalNumber analysed
Ravulizumab0 Participants58
Placebo (ECU-NMO-301)20 Participants47

Ravulizumab

0

Placebo (ECU-NMO-301)

20

p = < 0.0001; Hazard Ratio (HR) 0.014 (95% CI 0.000–0.103)

Values as posted on ClinicalTrials.gov. Outcomes measured differently across trials are not directly comparable.

Safety

GroupParticipants at riskSerious adverse eventsDeaths
Primary Treatment Period: Placebo4726 (55.3%)0
Primary Treatment Period: Ravulizumab588 (13.8%)0
Long-Term Extension: Ravulizumab569 (16.1%)1

As posted on ClinicalTrials.gov. Serious adverse events include any serious health event during the trial, whether or not the treatment caused it.

What it measured

Main measures

  • Number of Participants With an Adjudicated On-trial Relapse in the Primary Treatment Period

    Baseline up to 2.25 years (end of the Primary Treatment Period)

    Relapses

  • Time to first adjudicated On-Trial Relapse and relapse risk reduction

    Relapses

Other measures (10)

  • Adjudicated On-trial Annualized Relapse Rate (ARR) in the Primary Treatment Period

    Baseline up to 2.25 years (end of the Primary Treatment Period)

    Relapses

  • Number of Participants With Clinically Important Change From Baseline in Hauser Ambulation Index (HAI) Score at the End of Primary Treatment Period

    Baseline up to 2.25 years (end of the Primary Treatment Period)

    Walking and mobility

  • Change From Baseline in European Quality of Life Health 5-dimension Questionnaire (EQ-5D) Index Score at the End of Primary Treatment Period

    Baseline, up to 2.25 years (end of the Primary Treatment Period)

    Walking and mobilityPainMood and sleepQuality of life (reported by participants)

  • Change From Baseline in EQ-5D Visual Analog Scale (VAS) Score at the End of Primary Treatment Period

    Baseline, up to 2.25 years (end of the Primary Treatment Period)

    Quality of life (reported by participants)

  • Number of Participants With Clinically Important Worsening From Baseline in Expanded Disability Status Scale (EDSS) Score at the End of Primary Treatment Period

    Baseline up to 2.25 years (end of the Primary Treatment Period)

    Disability progression

  • 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

    Baseline up to 2.25 years (end of the Primary Treatment Period)

    Safety

  • Serum Ravulizumab Concentration

    Predose and end of infusion (EOI) at Week 26

  • Change From Baseline in Serum Free C5 Concentration at Week 26

    Baseline, Week 26 (Predose and EOI)

  • Number of Participants With Anti-drug Antibodies (ADAs) During the Primary Treatment Period

    Baseline, Weeks 26, 50, 82, and 106

    Immune response

  • 1. Adjudicated On Trial ARR

    Relapses

Who could take part

NMOSD · 18 to 100

Inclusion Criteria: 1. Anti-aquaporin-4 antibody-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria. 2. At least 1 attack or relapse in the last 12 months prior to the Screening Period. 3. Expanded Disability Status Scale score ≤7. 4. Participants who enter the study receiving supportive immunosuppressive therapy must be on a stable dosing regimen of adequate duration prior to Screening. 5. Body weight ≥40 kilograms. 6. 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: 1. History of Neisseria meningitidis infection. 2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer). 3. Previously or currently treated with a complement inhibitor. 4. Use of rituximab or mitoxantrone within 3 months prior to Screening. 5. Use of IV immunoglobulin within 3 weeks prior to Screening.

As registered. Criteria can change, so ask the trial team or your neurologist.

About this trial

In the sponsor's words

The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.

Papers

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History

    1. Started

      Dec 2019

    2. Main results due

      Mar 2022

    3. Finished

      Oct 2024

    4. Results posted

      Aug 2023

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