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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
21
Participants will receive eculizumab by intravenous (IV) infusion for 52 weeks.
Research Site
Beijing, China
Research Site
Dongguan, China
Research Site
Jinan, China
Research Site
Shanghai, China
The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).
The mean (95% Confidence Interval) of patient-level Adjudicated On-trial annualized relapse rate (ARR)
Time frame: Baseline through Week 52
The efficacy of eculizumab in anti-AQP4 antibody positive participants with NMOSD measured by Adjudicated On-trial annualized relapse rate (ARR).
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine
Time frame: Baseline through Week 52
The efficacy of eculizumab by assessing change from baseline to end of study in Expanded Disability Status Scale (EDSS) Score
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: The mean (95% Confidence Interval) change from baseline to end of study in EDSS score
Time frame: 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)
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: The mean (95% Confidence Interval) change from baseline to end of study in ambulatory function as measured by Hauser ambulation index (HAI)
Time frame: 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
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: The mean (95% Confidence Interval) change from baseline to end of study in EQ-5D index score
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Research Site
Taiyuan, China
Research Site
Wenzhou, China
Research Site
Wuhan, China
Time frame: Baseline through Week 52
Change From Baseline to end of study in EQ-5D Visual Analogue Scale (VAS) Score
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: The mean (95% Confidence Interval) change from baseline to end of study in EQ-5D VAS
Time frame: Baseline through Week 52
Serum Eculizumab Concentrations Over Time
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: Mean serum eculizumab concentrations at all scheduled visits
Time frame: Baseline through Week 52
Serum Free Complement Component 5 (C5) Concentrations Over Time
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: Mean changes in serum free C5 concentrations at all scheduled visits
Time frame: Baseline through Week 52
Number of Treatment-emergent Antidrug Antibody (ADA) Positive Participants
Intercurrent Event (ICE): * ICE1: Premature discontinuation of study intervention * ICE2: Initiation of disallowed therapy or medicine * Summary measure: Proportion of treatment-emergent ADA positive participants
Time frame: Baseline through Week 52
To characterize the overall safety of eculizumab in the treatment of NMOSD
Number of participants with TEAEs, SAEs, and TEAEs leading to study intervention discontinuation, with abnormal vital signs, abnormal ECG readings, and abnormal laboratory tests results
Time frame: Baseline through Week 52