BLAZE-Limiting Approach in NMOSD

Completed

• Conditions: Neuromyelitis Optica Spectrum Disorders (NMOSD)
• Interventions: Drug: Eculizumab administration

• Registration Number: NCT06888622
• Lead Sponsor: Huashan Hospital

Brief Summary

This is an observational cohort study based on data from the hospital-based NMOSD registry (Chinese Medical Research Registration Number MR-31-22-008563; ChiCTR2000030651). Between October 2023 (when eculizumab was approved for NMOSD in China) and February 2025, 26 consecutive patients with AQP4-IgG-positive NMOSD received eculizumab, and 9 of them were included in this study.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-10-01
• Status Verified: 2025-03
• Primary Completion: 2025-03-01
• Study Completion: 2025-03-03
• Study First Submitted: 2025-03-14
• Study First Submitted QC: 2025-03-20
• Last Update Submitted: 2025-03-20
• Study First Posted: 2025-03-21
• Last Update Posted: 2025-03-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

(1)Patients have been diagnosed with NMOSD and tested seropositive for AQP4 antibody; (2) Age ≥ 18 years; (3) Received eculizumab during an acute phase of NMOSD, defined as within 30 days of attack onset； (4) Adherence to an 8-week follow-up from eculizumab initiation.

Exclusion Criteria

1. Patients with unresolved Neisseria meningitidis infection or severe infections that preclude the use of immunotherapy;
2. Patients with severe comorbidities (such as heart failure, respiratory failure, severe hepatic or renal dysfunction, etc.);
3. Patients with incomplete records of clinical symptoms and signs, as well as insufficient data on serum marker tests in their medical records.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Eculizumab treatment group	Eculizumab administration	Eculizumab was administered intravenously at a dosage of 900 mg weekly for four consecutive weeks

Primary Outcome Measures

Name	Time	Method
The change in disability status measured by the MRC scale score	0, 1, 2, 3, 4, 8 weeks	Measured by the MRC scale score for patients with LETM
The change in disability status	0, 1, 2, 3, 4, 8 weeks	Measured by the best corrected visual acuity (BCVA)

Secondary Outcome Measures

Name	Time	Method
The Change in Opticospinal Impairment Scale (OSIS) scores	0, 1, 2, 3, 4, 8 weeks	Measured by Opticospinal Impairment Scale (OSIS) for the entire cohort.
Incidence of AEs and SAEs during eculizumab treatment	1, 2, 3, 4, 8 weeks	dverse events were coded using MedDRA version 27.1. Adverse events were analyzed in the safety analysis population (all patients who received at least one dose of study treatment) in terms of percentage incidence and as rates by exposure time (number of events per 100 patient-years of exposure and the associated 95% CI) to adjust for any differences in duration of exposure. The severity of adverse events was measured by NCI CTCAE version 5.0.
The Change in Expanded Disability Status Scale (EDSS) scores	0, 1, 2, 3, 4, 8 weeks	Measured by Expanded Disability Status Scale (EDSS) scores for the entire cohort.
The change in Visual Functional System Score (VFSS)	0, 1, 2, 3, 4, 8 weeks	Measured by Visual Functional System Score (VFSS) for patients with ON.

Trial Locations

Locations (1)

Huashan Hospital; 🇨🇳 Shanghai, Shanghai, China