Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Enrollment
- 11
- Locations
- 1
- Primary Endpoint
- The number of relapses
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Basic Science
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male or female patients ≥ 18 years old;
- •Diagnosis of NMO or NMO spectrum disorder according to the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria;
- •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;
- •Expanded disability status scale (EDSS) score ≤ 6.0;
- •Patients were seropositive for AQP4-IgG;
- •Able and willing to give written informed consent and comply with the requirements of the study protocol.
Exclusion Criteria
- •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);
- •Participation in another interventional study within the last 3 months;
- •Tumor disease currently or within the last 5 years;
- •Pregnancy, breastfeeding, or child-bearing potential during the course of the study;
- •Patients with clinically relevant heart, liver, kidney or bone marrow dysfunction;
- •History of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.
Arms & Interventions
Baricitinib
Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96.
Intervention: Baricitinib (Drug)
Outcomes
Primary Outcomes
The number of relapses
Time Frame: 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 Outcomes
- Changes in EDSS scores(Changes in EDSS from baseline to 96 weeks)
- 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)
- Changes in the number of peripheral blood B cell subsets(From baseline to 96 weeks)
- Changes in serum AQP4-IgG titer(From baseline to 96 weeks)
- Incidence of treatment-emergent adverse events [safety and tolerability](From baseline to 96 weeks)
Investigators
Qiang Liu
Professor of Department of Neurology
Tianjin Medical University General Hospital