Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

Sponsor

Tianjin Medical University General Hospital (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05792462

Collaborator

(none)

Enrollment

Location

Arm

18.1

Anticipated Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
NMO Spectrum Disorder	Drug: Baricitinib	Phase 1/Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Basic Science

Official Title:

Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders

Anticipated Study Start Date :

Mar 20, 2023

Anticipated Primary Completion Date :

Mar 20, 2024

Anticipated Study Completion Date :

Sep 20, 2024

Arms and Interventions

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.

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.

Outcome Measures

Primary Outcome Measures

First time to relapse [From 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

Worsening in EDSS [Worsening from baseline in EDSS 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.
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
Counts of peripheral blood B cell subsets [From baseline to 52 weeks]
Compare peripheral blood plasma cells before and one year after initial intervention
Determination of serum AQP4 antibodies [From baseline to 52 weeks]
Compare serum AQP4-ab titers before and one year after initial intervention
Incidence of treatment-emergent adverse events [safety and tolerability] [From baseline to 52 weeks]
Adverse events related to belimumab are recorded

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female patients ≥ 18 years old;
Diagnosis of NMO or NMO spectrum disorder according to the 2015 International diagnostic criteria for neuromyelitis optic;
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;
EDSS <=6.0;
Able and willing to give written informed consent and comply with the requirements of the study protocol.

Exclusion Criteria:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tianjin Medical University General Hospital	Tianjin	Tianjin	China	300052

Sponsors and Collaborators

Tianjin Medical University General Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Qiang Liu, Professor of Department of Neurology, Tianjin Medical University General Hospital

ClinicalTrials.gov Identifier:

NCT05792462

Other Study ID Numbers:

IRB2023-YX-012-01

First Posted:

Mar 31, 2023

Last Update Posted:

Mar 31, 2023

Last Verified:

Mar 1, 2023

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Neuromyelitis Optica

Study Results

No Results Posted as of Mar 31, 2023