Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD

NCT06724809 | Active Not Recruiting Phase 3

• 1 other identifier: D7412C00002
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 7

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).

Conditions

NMOSD; Neuromyelitis Optica Spectrum Disorders

Keywords

NMOSD; Neuromyelitis Optica Spectrum Disorders; eculizumab; anti-AQP4

Outcome Measures

Primary Outcomes (2)

• 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)

  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

  Baseline through Week 52

Secondary Outcomes (8)

• 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

Study Arms (1)

eculizumab

EXPERIMENTAL

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.

Drug: eculizumab

Interventions

eculizumab DRUG

Participants will receive eculizumab by intravenous (IV) infusion for 52 weeks.

eculizumab

Eligibility Criteria

• Age: 18 Years - 130 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Agree to use a male condom when having sexual intercourse with a WOCBP who is not currently pregnant.

You may not qualify if:

• 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
• Participation in any other investigational drug study or exposure to an investigational drug or device within 5 half-lives of treatment (if known) or 30 days, which is longer, before the first dose administration

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• AstraZeneca: collaborator

Study Sites (7)

Research Site

Beijing, 100016, China

Location

Research Site

Dongguan, 523059, China

Location

Research Site

Jinan, 250012, China

Location

Research Site

Shanghai, 200040, China

Location

Research Site

Taiyuan, 030001, China

Location

Research Site

Wenzhou, 325000, China

Location

Research Site

Wuhan, 430030, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Xiangjun Chen, M.D

  Huashan Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: December 9, 2024
• Study Start: January 16, 2025
• Primary Completion (Estimated): December 7, 2026
• Study Completion (Estimated): December 7, 2026
• Last Updated: December 19, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• Access Criteria: When a request has been approved AstraZeneca will provide access to the deidentified individual patient-level data in an approved sponsored tool. Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Locations

CN (7)