Eculizumab for Acute Attack of Neuromyelitis Optica Spectrum Disorder
- Conditions
- Neuromyelitis Optica Spectrum Disorder Attack
- Interventions
- Drug: IVMPDrug: Complement protein C5 inhibitor
- Registration Number
- NCT06673394
- Lead Sponsor
- Tianjin Medical University General Hospital
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 75
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description IVMP arm IVMP IVMP arm: 1000mg methylprednisolone x5d, oral prednisone 60mg, 5mg weekly decline + antibiotics eculizumab arm Complement protein C5 inhibitor 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). 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 eculizumab arm 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). 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
- Primary Outcome Measures
Name Time Method Mean change in OSIS from presentation to Day 28 Acute attack to week4 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. The OSIS evaluates four primary functions: Visual Acuity (VA) (0-8), Motor Function (0-7), Sensory Function (0-5), and Sphincter Function (0-5). A higher OSIS score indicates a more severe level of disability. The standards for the OSIS scoring are as follows:
Optic-Spinal Impairment Score (OSIS) Visual Acuity (VA) 0 Normal
1. Scotoma but VA (corrected) better than 20/30
2. VA 20/30-20/59
3. VA 20/60-20/100
4. VA 20/I0I-2012004
5. VA 20/20I-20/800.
6. Count fingers only
7. Light perception only
8. No light perception Motor Function
0 Normal
1. Abnormal signs (hyperreflexia, Babinski sign) without weakness
2. Mild weakness (MRC grade 5-or 4+) in affected limb(s)
3. Mo
- Secondary Outcome Measures
Name Time Method Change in EDSS from presentation to Day 28 Acute attack to week4 The Kurtzke Expanded Disability Status Scale (EDSS) 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. The EDSS provides a total score on a scale that ranges from 0 to 10 where 0 is normal and 10 is deceased. Increasing disability is reflected in an increasing EDSS score.