Eculizumab For the Acute Attack of Neuromyelitis Optica Spectrum Disorder

NCT07184840 | Recruiting Phase 2

• 1 other identifier: IRB2024-YX-456-01
• Study Type: interventional
• Target: 110
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Neuromyelitis Optica Spectrum Disorder Attack

Keywords

Eculizumab; Acute Attack; Neuromyelitis Optica Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Proportion of improvement in OSIS from baseline to Day 28 by 2 points or more

  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.

  Acute attack to Day 28

Secondary Outcomes (13)

• Change of OSIS from baseline to Day 28

  Acute attack to Day 28

• Change of EDSS from baseline to Day 28

  Acute attack to Day 28

• Proportion of subjects that require plasma exchange or immunoadsorption at day 10 post treatment initiation.

  Acute attack to Day 10

• Change of OSIS from baseline to Day 7, 14, 21, and Week 12.

  Acute attack to Week 12

• Change of EDSS from baseline to Day 7, 14, 21, and Week 12

  Acute attack to Week 12

Study Arms (2)

Intravenous Methylprednisolone (IVMP) arm

OTHER

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

Drug: Intravenous Methylprednisolone (IVMP)

IVMP+Eculizumab arm

EXPERIMENTAL

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.

Drug: Intravenous Methylprednisolone (IVMP); Drug: Complement protein C5 inhibitor

Interventions

Intravenous Methylprednisolone (IVMP) DRUG

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

IVMP+Eculizumab arm; Intravenous Methylprednisolone (IVMP) arm

Complement protein C5 inhibitor DRUG

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.

Also known as: Eculizumab

IVMP+Eculizumab arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Anti-AQP4 antibody seropositive.
• Male or female patients ≥18 years old
• Body weight ≥ 35 kg
• 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.
• 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

You may not qualify if:

• Use of IVIg within 3 weeks prior to screening
• Unresolved meningococcal infection
• Any systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics
• Participation in any other investigational drug study or was exposed to an investigational drug or device within 30 days of screening.
• Has previously received treatment with eculizumab
• Hypersensitivity to murine proteins or to one of the excipients of eculizumab
• 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

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead

Study Sites (1)

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, 300052, China

RECRUITING

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Fu-Dong Shi

CONTACT

+86-022-60814587; fshi@tmu.edu.cn

Wei Jiang

CONTACT

+86-022-60814587; jiangwei.med@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 8, 2025
• First Posted: September 22, 2025
• Study Start: September 9, 2025
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)