This is an open-label study, to evaluate the efficacy and safety of a BTK inhibitor zanubrutinib in participants with NMOSDs.
This is an open, single-center clinical study to evaluate the efficacy and safety of Zanubrutinib in the treatment of recurrent neuromyelitis optica spectrum disease. Patients were required to be diagnosed with neuromyelitis optic spectrum disease according to the NMOSD diagnostic criteria established by the international NMO Diagnostic Group (IPND) in 2015, and to have had at least two relapses (including first episode) within two years while at least one relapse occurring within the 12 months prior to screening. The AQP4 antibody must be positive during screening.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
zanubrutinib orally, 80mg bid for 1 year
XuanWu Hospital
Beijing, Beijing Municipality, China
RECRUITINGRelapse-free rate at week 48
Proportion of subjects who are relapse-free at week 48
Time frame: up to week 48
Changes in the expanded disability status scale (EDSS) score from baseline at week 12,24,36 and 48
EDSS is an ordered scale with values ranging from 0 points (normal neurological examination) to 10 points (death) in increments of 0.5 points. Higher scores indicate increased disability, and negative changes from baseline indicate improvement
Time frame: up to week12,24,36 and 48
Changes in visual acuity at week 12, 24,36 and 48 from baseline
Changes in visual acuity at week 12, 24,36 and 48 from baseline
Time frame: up to week12,24,36 and 48
Changes in the MOS item short from health survey (SF-36) score at week 12, 24,36 and 48 from baseline
The SF-36 is a multi-purpose, concise 36-question health survey scale. It contains 8 dimensions,The eight dimensions were converted to a scale of 0-100, with a higher score indicating a better quality of life. Positive change from baseline indicates improvement.
Time frame: up to week12,24,36 and 48
Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline
Changes in Serum AQP4-IgG titer at week 12, 24,36 and 48 from baseline
Time frame: up to week12,24,36 and 48
Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline
Changes in Peripheral blood lymphocyte subset at week 12, 24,36 and 48 from baseline
Time frame: up to week12,24,36 and 48
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Plasma concentraion of zanubrutinib
Concentraion of zanubrutinib in peripheral blood plasma
Time frame: up to week 48
Number of participants with all grade Adverse Events, Serious Adverse Events and who discontinued study therapy due to AEs
Evaluate Number of participants with all grade Adverse Events and Serious Adverse Events determined by NCI-CTCAE V5.0,and tolerance base on percentage of patients who discontinued study therapy due to AEs
Time frame: up to week 48