NIPH Clinical Trials Search

Safety and Efficacy Study of Sivelestat in Neuromyelitis Optica Spectrum Disorder

Basic Information

Recruitment status	Pending
Health condition(s) or Problem(s) studied	Neuromyelitis Optica Spectrum Disorder (NMOSD)
Date of first enrollment	01/08/2026
Target sample size	7
Countries of recruitment
Study type	Interventional
Intervention(s)	After dissolving sivelestat 100 mg in 10 mL of physiological saline, the daily dose (4.8 mg/kg as sivelestat sodium hydrate) will be diluted in 250-500 mL of physiological saline and administered by continuous intravenous infusion over 24 hours (0.2 mg/kg per hour) for 5 days. Additionally, steroid pulse therapy will be co-administered for all cases.

Outcome(s)

Primary Outcome

Incidence of adverse events

Secondary Outcome

1. Change from baseline in body temperature, blood pressure, pulse rate, and percutaneous arterial oxygen saturation at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration. 2. Change from baseline in each clinical laboratory value at Day 3, Day 6, and Day 28. 3. Change from baseline in EDSS at 4 weeks (Day 28). 4. Proportion of cases where EDSS improved by 1 point or more from baseline at 4 weeks (Day 28). 5. Change from baseline in Functional System (FS) domain scores at 4 weeks. 6. Change from baseline in OSIS at 4 weeks (Day 28). 7. Proportion of cases where EDSS score and OSIS improved to pre-relapse levels at 4 weeks (Day 28). 8. (For patients with optic neuritis as a relapse symptom) Change from baseline in the following ophthalmological examinations at 4 weeks (Day 28): i. Visual acuity ii. Thickness of peripapillary retinal nerve fiber layer and macular ganglion cell inner plexiform layer measured by OCT iii. Critical flicker fusion frequency 9. Proportion of cases with gadolinium-enhancing lesions on MRI at 4 weeks. 10. Proportion of cases requiring a second course of steroid pulse therapy, plasmapheresis, or high-dose immunoglobulin therapy.

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	Not applicable
Gender	Both
Include criteria	1.Patients diagnosed with anti-AQP4 antibody-positive NMOSD according to the international diagnostic criteria for NMOSD (Wingerchuk, Neurology 2015). 2.Patients experiencing a relapse including any of the following, with the relapse occurring within 14 days of obtaining consent: i. Unilateral or bilateral optic neuritis ii. Myelitis 3. Patients whose FS domain has worsened by at least 1 point due to relapse. 4. Patients with one or more relapse lesions identified on MRI (however, if the relapse is considered to have occurred in the same location as an existing MRI lesion neurologically, identification of a new relapse lesion is not required). 5. Patients aged 18 years or older at the time of obtaining consent. 6. Female patients of childbearing potential who agree to use appropriate contraception from the time of obtaining consent until 180 days after the end of investigational product administration. 7.Male patients who agree to use appropriate contraception until 90 days after the end of investigational product administration. 8. Patients who can provide written informed consent.
Exclude criteria	1. Patients with multi-organ dysfunction involving 4 or more organs. 2. Patients with severe chronic respiratory disease. 3. Patients with autoimmune diseases other than NMOSD that are expected to require additional treatment during the study period. 4. Patients with active systemic bacterial, viral, or fungal infections. 5. Patients who have received either or both of the following prior treatments after an NMOSD relapse: i. Two or more courses of steroid pulse therapy ii. Plasmapheresis iii. High-dose immunoglobulin therapy 6. Patients with severe hepatic dysfunction. 7. Patients with alcohol dependence, drug dependence, or psychiatric disorders that would interfere with study participation. 8. Patients who have received other investigational drugs within 3 months prior to obtaining consent. 9. Pregnant women, women suspected of being pregnant, or breastfeeding women. 10. Patients with allergies to the investigational product or concomitant medications. 11. Patients with severe allergies or a history of severe allergies. 12. Patients with suicidal tendencies meeting any of the following criteria: i. Within 1 month prior to the screening assessment, there was suicidal behavior or ideation corresponding to "Yes" for Item 4 (Active suicidal ideation -some intent to act, but no specific plan) or Item 5 (Active suicidal ideation -specific plan and intent) of the Columbia-Suicide Severity Rating Scale (C-SSRS). (For subjects who only met Items 1-3, inclusion may be permitted at the discretion of the principal investigator or sub-investigator.) ii. Any suicidal behavior based on Item 6 of the C-SSRS occurred within the past 3 months. 13. Other patients judged inappropriate by the principal investigator or sub-investigator.