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

Study Overview

• Status: Active, not recruiting
• Conditions: NMO Spectrum Disorder
• Intervention / Treatment: Drug: Baricitinib

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 Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300052
      • Tianjin Medical University General Hospital

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 Panel for Neuromyelitis Optica Diagnosis criteria;
3. 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;
4. Expanded disability status scale (EDSS) score ≤ 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 study within the last 3 months;
3. Tumor disease currently or within the last 5 years;
4. Pregnancy, breastfeeding, or child-bearing potential during the course of the study;
5. Patients with clinically relevant heart, liver, kidney or bone marrow dysfunction;
6. History of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.

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 4mg once daily until the disease relapses or week 96.	Drug: Baricitinib; Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of relapses	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.	From baseline to 96 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in EDSS scores	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 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).	The total number of new and/or enlarging lesions on T2-weighted imaging (T2WI) and gadolinium-enhancing lesions on T1-weighted imaging (T1WI) for all participants was calculated as the sum of the individual number of lesions at Weeks 24, 48, and 96	From baseline to 96 weeks
Changes in the number of peripheral blood B cell subsets	Compare peripheral blood plasma cells before and two year after initial intervention	From baseline to 96 weeks
Changes in serum AQP4-IgG titer	Compare serum AQP4-IgG titers before and two year after initial intervention	From baseline to 96 weeks
Incidence of treatment-emergent adverse events [safety and tolerability]	Adverse events related to baricitinib are recorded	From baseline to 96 weeks

Collaborators and Investigators

• Sponsor: Tianjin Medical University General Hospital
• Collaborators: Tang-Du Hospital; The Second Hospital of Shandong University
• 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): March 1, 2027
• Study Completion (Estimated): March 1, 2027

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): August 11, 2026
• Last Update Submitted That Met QC Criteria: August 9, 2026
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica; baricitinib
• 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