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
No
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
More Tianjin Medical University General Hospital trials in China
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.
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