An Open-label, Single-arm, Multi-Center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adult Participants With Anti-Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorders (NMOSD)

Alexion Pharmaceuticals, Inc.1 site in 1 country21 target enrollmentJanuary 16, 2025

ConditionsNMOSD Neuromyelitis Optica Spectrum Disorders

Interventionseculizumab

Drugseculizumab

Overview

Phase: Phase 3
Intervention: eculizumab
Conditions: NMOSD
Sponsor: Alexion Pharmaceuticals, Inc.
Enrollment: 21
Locations: 1
Primary Endpoint: The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).
Status: Active, Not Recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).

Registry

clinicaltrials.gov

Start Date

January 16, 2025

End Date

December 7, 2026

Last Updated

4 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Alexion Pharmaceuticals, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Participants with diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria
•Anti-AQP4 antibody positive
•At least 1 attack or relapse in the last 12 months prior to the Screening Period
•EDSS score ≤ 7
•If a participant enters the study receiving IST(s) for relapse prevention, the participant must be on a stable maintenance dose of IST(s) as follows, prior to screening and must remain on that dose for the duration of the study, unless the participant experiences a relapse
•Female participants of childbearing potential must have a negative pregnancy test (serum HCG at screening
•Male participants are eligible to participate if they agree to the following during the study intervention Treatment Period and for at least 5 months after the last dose of study intervention:
•Refrain from donating fresh unwashed semen. PLUS, either,
•Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent. OR
•Must agree to use barrier as detailed below:

Exclusion Criteria

•Pregnant, breastfeeding, or intending to conceive during the course of the study
•Prior history of N meningitidis infection or unresolved meningococcal disease
•Any systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics
•Presence of fever ≥ 38 C within 7 days prior to study intervention administration on Day 1
•Hypersensitivity to murine proteins or to one of the excipients of study intervention
•Use of rituximab, inebilizumab, or other B cell-depleting therapy within 6 months prior to Day 1 and during the study
•Use of mitoxantrone or satralizumab within 3 months prior to screening and during the study
•Use of IVIg within 3 weeks prior to screening
•If a participant enters the study receiving oral corticosteroid(s) with or without other ISTs, the daily corticosteroid dose must be no more than prednisone 20 mg/day (or equivalent) prior to screening and the participant must remain on that dose for the duration of the study or until the participant experiences a relapse (specific medications listed in Section 6.9.1 may be allowed)
•Has previously received treatment with C5 inhibitors

Arms & Interventions

eculizumab

All participants will receive open-label eculizumab by intravenous infusion during the Treatment Period, starting on Day 1 for a total of up to 52 weeks.

Intervention: eculizumab

Outcomes

Primary Outcomes

The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).

Time Frame: Baseline through Week 52

Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine

Secondary Outcomes

The efficacy of eculizumab by assessing change from baseline to end of study in Expanded Disability Status Scale (EDSS) Score(Baseline through Week 52)
The efficacy of eculizumab by assessing change from baseline to end of study in Ambulatory Function as Measured by Hauser Ambulation Index (HAI)(Baseline through Week 52)
The efficacy of eculizumab by assessing change from baseline to end of study in European Quality of Life 5-Dimension Questionnaire (EQ-5D) Index Score(Baseline through Week 52)
Change From Baseline to end of study in EQ-5D Visual Analogue Scale (VAS) Score(Baseline through Week 52)
Serum Eculizumab Concentrations Over Time(Baseline through Week 52)
Serum Free Complement Component 5 (C5) Concentrations Over Time(Baseline through Week 52)
Number of Treatment-emergent Antidrug Antibody (ADA) Positive Participants(Baseline through Week 52)
To characterize the overall safety of eculizumab in the treatment of NMOSD(Baseline through Week 52)