NCT06673394

Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder

Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2, Open Label Trial (EASE-NMO)

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Tianjin Medical University General Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	Complement protein C5 inhibitor	Investigational 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). Terminal complement complex inhibition by eculizumab predisposes patients to infections by encapsulated bacteria, especially N meningitidis. Meningococcal vaccination will not be effective in this timeframe; instead, all enrolled patients will receive antibiotic prophylaxis against N meningitidis from the time of the first dose of the study drug to 8 weeks after the last administration.
DRUG	IVMP	IVMP arm: 1000mg methylprednisolone x5d, oral prednisone 60mg, 5mg weekly decline + antibiotics

Timeline

• Start date: 2025-09-03
• Primary completion: 2026-06-01
• Completion: 2026-12-01
• First posted: 2024-11-04
• Last updated: 2025-09-10