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

NCT ID: NCT06724809

Last Updated: 2025-12-19

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-16
• Study Completion Date: 2026-12-07

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

eculizumab

Intervention Type: DRUG

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

Interventions

eculizumab

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

Intervention Type: DRUG

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:

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 130 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AstraZeneca

INDUSTRY

Sponsor Role: collaborator

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Xiangjun Chen, M.D

Role: PRINCIPAL_INVESTIGATOR

Huashan Hospital

Locations

Research Site

Beijing, , China

Research Site

Dongguan, , China

Research Site

Jinan, , China

Research Site

Shanghai, , China

Research Site

Taiyuan, , China

Research Site

Wenzhou, , China

Research Site

Wuhan, , China

Countries

China

Other Identifiers

D7412C00002

Identifier Type: -

Identifier Source: org_study_id