Eculizumab For the Acute Attack of Neuromyelitis Optica Spectrum Disorder

NCT07184840 · tracked via the Priya Life Science China tracker

Sponsor

Tianjin Medical University General Hospital

Phase

Phase 2

Started

2025-09-09

Last updated

2026-06-03

Condition(s) studied

Neuromyelitis Optica Spectrum Disorder Attack

Investigational drug(s) / intervention(s)

Intravenous Methylprednisolone (IVMP) →Complement protein C5 inhibitor →

Intravenous Methylprednisolone (IVMP): IVMP arm: 1000mg methylprednisolone x5d, oral prednisone 60mg, 5mg weekly decline + antibiotics

Complement protein C5 inhibitor: IVMP+Eculizumab arm: eculizumab (900 mg) will be administered intravenously once per week for a total of four doses (days 1, 8, 15, and 22) in conjunction with IVMP and oral prednisone (60mg, 5mg weekly decline). All enrolled patients will receive antibiotic prophylaxis against N meningitidis.

Study summary

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: 1. Anti-AQP4 antibody seropositive. 2. Male or female patients ≥18 years old 3. Body weight ≥ 35 kg 4. Acute optic neuritis and/or transverse myelitis enrolled within 28 days from the attack, with a change in neurological exam that meet an increase of OSIS at least 2 points of baseline compared to that of prior attack. 5. A female subject is eligible to enter the trial if she is: * Not pregnant or breastfeeding, not intending to conceive during the course of the trial Exclusion Criteria: 1. Use of IVIg within 3 weeks prior to screening 2. Unresolved meningococcal infection 3. Any systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics 4. Participation in any other investigational drug study or was exposed to an investigational drug or device within 30 days of screening. 5. Has previously received treatment with eculizumab 6. Hypersensitivity to murine proteins or to one of the excipients of eculizumab 7. Any medical condition that, in the opinion of the Investigator, might interfere with thepatient's participation in the trial, poses any added risk for the patient, or confounds the assessment of the patients

Primary outcome measure(s)

Proportion of improvement in OSIS from baseline to Day 28 by 2 points or more — Acute attack to Day 28
The Optic-Spinal Impairment Score (OSIS) was developed to measure the disability status of subjects with demyelinating disease. It allows an objective quantification of the level of functioning that could be widely and reproducibly used by researchers and health care providers. OSIS score ranges from 0 to 25, which includes four primary functions: Visual Acuity (VA) (0-8), Motor Function (0-7), Sensory Function (0-5), and Sphincter Function (0-5). The higher scores reflect more severe disability. A decrease of at least 2 points in the overall OSIS score on day 28 compared to the baseline was regarded as a significant improvement.

Trial sites (1)

Facility	City	Region	Status
Tianjin Medical University General Hospital	Tianjin	Tianjin Municipality

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07184840 on ClinicalTrials.gov ↗ ← All trials in China