Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

NCT05792462 | Recruiting Phase 1

• 1 other identifier: IRB2023-YX-012-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

NMO Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• The number of attacks

  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

  From baseline to one year after

Secondary Outcomes (5)

• Changes in EDSS

  Changes in EDSS from baseline to 52 weeks

• 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

• Changes in peripheral blood B cell subsets

  From baseline to 52 weeks

• Changes in serum AQP4 antibodies

  From baseline to 52 weeks

• Incidence of treatment-emergent adverse events [safety and tolerability]

  From baseline to 52 weeks

Study Arms (1)

Baricitinib

EXPERIMENTAL

Baricitinib 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.

Drug: Baricitinib

Interventions

Baricitinib DRUG

Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 48, with a final evaluation at week 52.

Baricitinib

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 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;
• EDSS \<=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.

You may not qualify if:

• 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 trial within the last 3 months Tumor disease currently or within last 5 years;
• Pregnant, breastfeeding, or child-bearing potential during the course of the study Clinically relevant heart, liver, kidney or bone marrow function disorder.
• Have a history of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead
• Tang-Du Hospital: collaborator
• The Second Hospital of Shandong University: collaborator

Study Sites (1)

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, 300052, China

RECRUITING

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

baricitinib

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Qiang Liu, M.D.,PhD

  Tianjin Medical University General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Qiang Liu, M.D.,Ph.D.

CONTACT

+86 15022439149; qliu@tmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Department of Neurology

Study Record Dates

• First Submitted: March 18, 2023
• First Posted: March 31, 2023
• Study Start: April 15, 2023
• Primary Completion: December 30, 2025
• Study Completion: December 30, 2025
• Last Updated: October 16, 2024

Record last verified: 2024-10

Locations

CN (1)