AzTrials
AzTrials

NMOSDPhase 3ECU-NMO-304NCT06724809

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

A Phase 3 study testing eculizumab in people with NMOSD and Neuromyelitis Optica Spectrum Disorders, run by Alexion Pharmaceuticals, Inc. at 7 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 18 to 130 years
What is being tested?
eculizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
7 sitesChina
Study dates
16 Jan 2025to 7 Dec 2026
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 130 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 11Show fewer
  • 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.

Who cannot join

  • 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
Show all 11Show fewer
  • 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

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

China7 sites

  • Research Site

    Beijing, 100016

  • Research Site

    Dongguan, 523059

  • Research Site

    Jinan, 250012

  • Research Site

    Shanghai, 200040

  • Research Site

    Taiyuan, 030001

  • Research Site

    Wenzhou, 325000

  • Research Site

    Wuhan, 430030

Other trials in China

About this study

Official title: 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).

Being tested
eculizumab
Keywords
NMOSD, Neuromyelitis Optica Spectrum Disorders, eculizumab, anti-AQP4
Medical subject headings
Neuromyelitis Optica

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • eculizumabDrug

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

Study groups in NCT06724809
Study groupRoleWhat they receive
eculizumabExperimentalAll 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.
Receives: Drug: eculizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    Measured: Baseline through Week 52

  2. 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

    Measured: Baseline through Week 52

Secondary outcomes (8)

  1. 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

    Measured: Baseline through Week 52

  2. 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)

    Measured: Baseline through Week 52

  3. 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

    Measured: Baseline through Week 52

  4. 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

    Measured: Baseline through Week 52

  5. 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

    Measured: Baseline through Week 52

  6. 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

    Measured: Baseline through Week 52

  7. 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

    Measured: Baseline through Week 52

  8. 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

    Measured: Baseline through Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Dec 2024
  2. Study start16 Jan 2025
  3. Primary completion7 Dec 2026 (estimated)
  4. Study completion7 Dec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06724809
Sponsor's protocol ID
D7412C00002
Acronym
ECU-NMO-304
Status last verified
Dec 2025
First submitted
19 Nov 2024
Last updated
19 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing eculizumab in people with NMOSD and Neuromyelitis Optica Spectrum Disorders, run by Alexion Pharmaceuticals, Inc. at 7 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06724809.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in