BLAZE-Limiting Approach in NMOSD

Eculizumab for Blaze-limiting Approach in NMOSD: A Prospective Study

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.

Study Overview

• Status: Completed
• Conditions: Neuromyelitis Optica Spectrum Disorders (NMOSD)
• Intervention / Treatment: Drug: Eculizumab administration

• Study Type: Observational
• Enrollment (Actual): 9

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200040
      • Huashan Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Eculizumab treatment group; Eculizumab was administered intravenously at a dosage of 900 mg weekly for four consecutive weeks	Drug: Eculizumab administration; Eculizumab was administered intravenously at a dosage of 900 mg weekly for four consecutive weeks

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of AEs and SAEs during eculizumab treatment	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.	1, 2, 3, 4, 8 weeks
The Change in Expanded Disability Status Scale (EDSS) scores	Measured by Expanded Disability Status Scale (EDSS) scores for the entire cohort.	0, 1, 2, 3, 4, 8 weeks
The Change in Opticospinal Impairment Scale (OSIS) scores	Measured by Opticospinal Impairment Scale (OSIS) for the entire cohort.	0, 1, 2, 3, 4, 8 weeks
The change in Visual Functional System Score (VFSS)	Measured by Visual Functional System Score (VFSS) for patients with ON.	0, 1, 2, 3, 4, 8 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
The changes of serological biomarkers (sGFAP and sNfL)	Serum GFAP and NfL concentrations were analyzed in duplicates using SIMOA.	0, 2, 4, 8 weeks
The changes of OCT measurements (pRNFL and mGCIPL).	The peripapillary retinal nerve fiber layer (pRNFL) and macular ganglion cell-inner plexiform layer (mGCIPL) was analyzed by optical coherence tomography (OCT) measurements	0, 1, 2, 3, 4, 8 weeks

Collaborators and Investigators

• Sponsor: Huashan Hospital
• Investigators: Principal Investigator: Chao Quan, Doctor, Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, People's Republic of China

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2023
• Primary Completion (Actual): March 1, 2025
• Study Completion (Actual): March 3, 2025

Study Registration Dates

• First Submitted: March 14, 2025
• First Submitted That Met QC Criteria: March 20, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 20, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: eculizumab; Neuromyelitis Optica Spectrum Disorders; NMOSD; AQP4-ab
• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; Eculizumab
• Other Study ID Numbers: KY2025-062; MR-31-22-008563; ChiCTR2000030 (Other Identifier: Chinese Clinical Trial Registry)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No