NMO Spectrum Disorder
Conditions
Brief summary
Neuromyelitis Optica Spectrum Disorders (NMOSD) is associated with a pathological humoral immune response against the aquaporin-4(AQP-4) water channel. Baricitinib is an oral Janus kinase (JAK)1/JAK2 inhibitor that blocks the upregulated JAK-STAT pathway in patients with neuroimmune disorders, which is important in bone marrow regulation of B cell proliferation and differentiation. Baricitinib may benefit some patients with NMOSD due to the important role of B cells in the pathogenesis of NMOSD. Clinical trials may be needed to observe its efficacy and safety.
Detailed description
The investigators primarily aim to observe the number of relapses from initiation of baricitinib treatment. The secondary outcomes are to determine: The safety profile of baricitinib in participants with NMO and whether baricitinib improves Expanded Disability Status Scale (EDSS), et al.
Interventions
DRUGBaricitinib
Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96.
Sponsors
Tianjin Medical University General Hospital
Tang-Du Hospital
The Second Hospital of Shandong University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male or female patients ≥ 18 years old; 2. Diagnosis of NMO or NMO spectrum disorder according to the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria; 3. Clinical evidence of either at least one relapse requiring rescue therapy (intravenous corticosteroids, intravenous immunoglobulin, plasma exchange or a combination of these therapies) in the year before screening or at least two relapses requiring rescue therapy in the 2 years before screening; 4. Expanded disability status scale (EDSS) score ≤ 6.0; 5. Patients were seropositive for AQP4-IgG; 6. Able and willing to give written informed consent and comply with the requirements of the study protocol.
Exclusion criteria
1. Current evidence or known history of clinically significant infection (Herpes simplex virus, varicella-zoster virus, cytomegalovirus, Epstein-Barr virus, human immunodeficiency virus, Hepatitis viruses, Syphilis, etc); 2. Participation in another interventional study within the last 3 months; 3. Tumor disease currently or within the last 5 years; 4. Pregnancy, breastfeeding, or child-bearing potential during the course of the study; 5. Patients with clinically relevant heart, liver, kidney or bone marrow dysfunction; 6. History of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The number of relapses | From baseline to 96 weeks | A relapse was defined as new-onset neurological symptoms or worsening of existing neurological function (vision loss, limb weakness or sensory symptoms, or bladder or bowel dysfunction) lasting more than 24 h, not attributable to an identifiable cause such as intercurrent infection, and preceded by at least 30 days of clinical stability. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in EDSS scores | Changes in EDSS from baseline to 96 weeks | The Expanded Disability Status Scale (EDSS) is a rating system that is frequently used for classifying and standardizing the severity and progression. EDSS ranges from 0 to 10. |
| Changes in the number of new and/or enlarging lesions on T2-weighted imaging (T2WI) and gadolinium-enhancing lesions on T1-weighted imaging (T1WI). | From baseline to 96 weeks | The total number of new and/or enlarging lesions on T2-weighted imaging (T2WI) and gadolinium-enhancing lesions on T1-weighted imaging (T1WI) for all participants was calculated as the sum of the individual number of lesions at Weeks 24, 48, and 96 |
| Changes in the number of peripheral blood B cell subsets | From baseline to 96 weeks | Compare peripheral blood plasma cells before and two year after initial intervention |
| Changes in serum AQP4-IgG titer | From baseline to 96 weeks | Compare serum AQP4-IgG titers before and two year after initial intervention |
| Incidence of treatment-emergent adverse events [safety and tolerability] | From baseline to 96 weeks | Adverse events related to baricitinib are recorded |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORQiang Liu, M.D.,PhD
Tianjin Medical University General Hospital
Outcome results
None listed