Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD (ECU-NMO-304)

December 15, 2025 updated by: Alexion Pharmaceuticals, Inc.

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)

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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
      • Beijing, China, 100016
        • Research Site
      • Dongguan, China, 523059
        • Research Site
      • Jinan, China, 250012
        • Research Site
      • Shanghai, China, 200040
        • Research Site
      • Taiyuan, China, 030001
        • Research Site
      • Wenzhou, China, 325000
        • Research Site
      • Wuhan, China, 430030
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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:

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

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
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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.
Drug: eculizumab
Participants will receive eculizumab by intravenous (IV) infusion for 52 weeks.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
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).
Time Frame: Baseline through Week 52

Intercurrent Event (ICE):

  • ICE1: Premature discontinuation of study intervention
  • ICE2: Initiation of disallowed therapy or medicine
Baseline through Week 52

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
The efficacy of eculizumab by assessing change from baseline to end of study in Expanded Disability Status Scale (EDSS) Score
Time Frame: Baseline through Week 52

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
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)
Time Frame: Baseline through Week 52

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)
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
Time Frame: Baseline through Week 52

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
Baseline through Week 52
Change From Baseline to end of study in EQ-5D Visual Analogue Scale (VAS) Score
Time Frame: Baseline through Week 52

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
Baseline through Week 52
Serum Eculizumab Concentrations Over Time
Time Frame: Baseline through Week 52

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
Baseline through Week 52
Serum Free Complement Component 5 (C5) Concentrations Over Time
Time Frame: Baseline through Week 52

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
Baseline through Week 52
Number of Treatment-emergent Antidrug Antibody (ADA) Positive Participants
Time Frame: Baseline through Week 52

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
Baseline through Week 52
To characterize the overall safety of eculizumab in the treatment of NMOSD
Time Frame: Baseline through Week 52
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
Baseline through Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
AstraZeneca

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Xiangjun Chen, M.D, Huashan Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 16, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 7, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 7, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 19, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 5, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 9, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 19, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 15, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • D7412C00002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal.

All request will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

IPD Sharing 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.

IPD Sharing 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.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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