Clinical trial NCT05792462: Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Official title: Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: NMO Spectrum Disorder
Lead sponsor: Tianjin Medical University General Hospital
Sponsor class: OTHER
Collaborators: name: Tang-Du Hospital
name: The Second Hospital of Shandong University
First posted: 2023-03-31
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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 attacks 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.

Eligibility criteria: 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 attack requiring rescue therapy (intravenous corticosteroids, intravenous immunoglobulin, plasma exchange, or a combination of these therapies) in the year before screening or at least two attacks requiring rescue therapy in the 2 years before screening;
4. EDSS \<=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 trial within the last 3 months Tumor disease currently or within last 5 years;
3. Pregnant, breastfeeding, or child-bearing potential during the course of the study Clinically relevant heart, liver, kidney or bone marrow function disorder.
4. Have a history of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.

Primary outcomes:
measure: The number of attacks
timeFrame: From baseline to one year after
Secondary outcomes:
measure: Changes in EDSS
timeFrame: Changes in EDSS from baseline to 52 weeks
measure: Changes in the number of New, and/or Enlarging T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)
timeFrame: From baseline to 52 weeks
measure: Changes in peripheral blood B cell subsets
timeFrame: From baseline to 52 weeks
measure: Changes in serum AQP4 antibodies
timeFrame: From baseline to 52 weeks
measure: Incidence of treatment-emergent adverse events [safety and tolerability]
timeFrame: From baseline to 52 weeks
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
state: Tianjin
Study officials:
affiliation: Tianjin Medical University General Hospital
name: Qiang Liu, M.D.,PhD
role: PRINCIPAL_INVESTIGATOR
References: