Clinical trial NCT06888622: BLAZE-Limiting Approach in NMOSD
Official title: Eculizumab for Blaze-limiting Approach in NMOSD: A Prospective Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Neuromyelitis Optica Spectrum Disorders (NMOSD)
Keywords: NMOSD
eculizumab
AQP4-ab
Neuromyelitis Optica Spectrum Disorders
Lead sponsor: Huashan Hospital
Sponsor class: OTHER
First posted: 2025-03-21
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This is an observational cohort study based on data from the prospectively followed NMOSD cohort of the hospital-based registry (The Chinese Medical Research Registration Number MR-31-22-008563; ChiCTR2000030651). Between October 2023 (when eculizumab was approved for NMOSD in China) and February 2025 consecutive patients with AQP4-IgG-positive NMOSD received eculizumab were enrolled.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: The change in disability status measured by the MRC scale score
timeFrame: 0, 1, 2, 3, 4, 8 weeks
measure: The change in disability status
timeFrame: 0, 1, 2, 3, 4, 8 weeks
Secondary outcomes:
measure: Incidence of AEs and SAEs during eculizumab treatment
timeFrame: 1, 2, 3, 4, 8 weeks
measure: The Change in Expanded Disability Status Scale (EDSS) scores
timeFrame: 0, 1, 2, 3, 4, 8 weeks
measure: The Change in Opticospinal Impairment Scale (OSIS) scores
timeFrame: 0, 1, 2, 3, 4, 8 weeks
measure: The change in Visual Functional System Score (VFSS)
timeFrame: 0, 1, 2, 3, 4, 8 weeks
Other outcomes:
measure: The changes of serological biomarkers (sGFAP and sNfL)
timeFrame: 0, 2, 4, 8 weeks
measure: The changes of OCT measurements (pRNFL and mGCIPL).
timeFrame: 0, 1, 2, 3, 4, 8 weeks
Locations:
city: Shanghai
country: China
facility: Huashan Hospital
state: Shanghai
Study officials:
affiliation: Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, People's Republic of China
name: Chao Quan, Doctor
role: PRINCIPAL_INVESTIGATOR
References: