Back to resultsEfficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Primary Purpose
NMO Spectrum Disorder
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Baricitinib
Sponsored by
About this trial
This is an interventional basic science trial for NMO Spectrum Disorder
Eligibility Criteria
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.
Sites / Locations
- Tianjin Medical University General HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Baricitinib
Arm Description
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.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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 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
Changes in peripheral blood B cell subsets
Compare peripheral blood plasma cells before and one year after initial intervention
Changes in serum AQP4 antibodies
Compare serum AQP4-ab titers before and one year after initial intervention
Incidence of treatment-emergent adverse events [safety and tolerability]
Adverse events related to belimumab are recorded
Full Information
NCT ID
NCT05792462
First Posted
March 18, 2023
Last Updated
June 10, 2023
Sponsor
Tianjin Medical University General Hospital
1. Study Identification
Unique Protocol Identification Number
NCT05792462
Brief Title
Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Official Title
Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 15, 2023 (Actual)
Primary Completion Date
March 20, 2024 (Anticipated)
Study Completion Date
September 20, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Tianjin Medical University General Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
NMO Spectrum Disorder
7. Study Design
Primary Purpose
Basic Science
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Baricitinib
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Baricitinib
Intervention Description
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.
Primary Outcome Measure Information:
Title
First time to relapse
Description
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
Time Frame
From baseline to one year after
Secondary Outcome Measure Information:
Title
Changes in EDSS
Description
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.
Time Frame
Changes in EDSS from baseline to 52 weeks
Title
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)
Description
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
Time Frame
From baseline to 52 weeks
Title
Changes in peripheral blood B cell subsets
Description
Compare peripheral blood plasma cells before and one year after initial intervention
Time Frame
From baseline to 52 weeks
Title
Changes in serum AQP4 antibodies
Description
Compare serum AQP4-ab titers before and one year after initial intervention
Time Frame
From baseline to 52 weeks
Title
Incidence of treatment-emergent adverse events [safety and tolerability]
Description
Adverse events related to belimumab are recorded
Time Frame
From baseline to 52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Qiang Liu, M.D.,Ph.D.
Phone
+86 15022439149
Email
qliu@tmu.edu.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Qiang Liu, M.D.,PhD
Organizational Affiliation
Tianjin Medical University General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Tianjin Medical University General Hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300052
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Qiang Liu, M.D.,Ph.D.
Phone
+8615022439149
Email
qliu@tmu.edu.cn
First Name & Middle Initial & Last Name & Degree
Qiang Liu, M.D.,Ph.D.
12. IPD Sharing Statement
Learn more about this trial
Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
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