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Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

Phase 1
Recruiting
Conditions
NMO Spectrum Disorder
Interventions
Registration Number
NCT05792462
Lead Sponsor
Tianjin Medical University General Hospital
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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
12
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
BaricitinibBaricitinibBaricitinib will be taken orally with a dose of 2mg once daily until the disease relapses or week 48, with a final evaluation at week 52.
Primary Outcome Measures
NameTimeMethod
The number of attacksFrom baseline to one year after

An acute attack was defined as a new neurological worsening lasting for at least 24 hours and occurring more than 30 days after the previous attack

Secondary Outcome Measures
NameTimeMethod
Changes in serum AQP4 antibodiesFrom baseline to 52 weeks

Compare serum AQP4-ab titers before and one year after initial intervention

Changes in peripheral blood B cell subsetsFrom baseline to 52 weeks

Compare peripheral blood plasma cells before and one year after initial intervention

Changes in EDSSChanges in EDSS from baseline to 52 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 T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)From baseline to 52 weeks

The total number of new and/or enlarging T2 lesions for all participants was calculated as the sum of the individual number of lesions at Weeks 12, 24, and 52

Incidence of treatment-emergent adverse events [safety and tolerability]From baseline to 52 weeks

Adverse events related to belimumab are recorded

Trial Locations

Locations (1)

Tianjin Medical University General Hospital

🇨🇳

Tianjin, Tianjin, China

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