Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

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. Clincial trials may be needed to observe its efficacy and safety.

Study Overview

• Status: Recruiting
• Conditions: NMO Spectrum Disorder
• Intervention / Treatment: Drug: Baricitinib

Detailed Description

The investigators primarily aim to observe the time to first relapse 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 Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Qiang Liu, M.D.,Ph.D.; Phone Number: +86 15022439149; Email: qliu@tmu.edu.cn

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300052
      • Recruiting
      • Tianjin Medical University General Hospital
      • Contact: Qiang Liu, M.D.,Ph.D.; Phone Number: +8615022439149; Email: qliu@tmu.edu.cn
      • Principal Investigator: Qiang Liu, M.D.,Ph.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female patients ≥ 18 years old;
2. Diagnosis of NMO or NMO spectrum disorder according to the 2015 International diagnostic criteria for neuromyelitis optic;
3. Clinical evidence of either at least one attack requiring rescue therapy (intravenous corticosteroids,intravenous immunoglobulin,plasma exchange,or a combination of these therapies) or at least two attacks requiring rescue therapy in the 2 years before screening;
4. EDSS <=6.0;
5. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Baricitinib; 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; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
First time to relapse	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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events [safety and tolerability]	Adverse events related to belimumab are recorded	From baseline to 52 weeks
Changes in EDSS	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 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)	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	From baseline to 52 weeks
Changes in peripheral blood B cell subsets	Compare peripheral blood plasma cells before and one year after initial intervention	From baseline to 52 weeks
Changes in serum AQP4 antibodies	Compare serum AQP4-ab titers before and one year after initial intervention	From baseline to 52 weeks

Collaborators and Investigators

• Sponsor: Tianjin Medical University General Hospital
• Investigators: Principal Investigator: Qiang Liu, M.D.,PhD, Tianjin Medical University General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2023
• Primary Completion (Estimated): December 30, 2025
• Study Completion (Estimated): December 30, 2025

Study Registration Dates

• First Submitted: March 18, 2023
• First Submitted That Met QC Criteria: March 18, 2023
• First Posted (Actual): March 31, 2023

Study Record Updates

• Last Update Posted (Actual): April 10, 2024
• Last Update Submitted That Met QC Criteria: April 9, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Myelitis, Transverse; Optic Neuritis; Neuromyelitis Optica
• Other Study ID Numbers: IRB2023-YX-012-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No