NMO Spectrum DisorderPhase 1NCT05792462
Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders
A Phase 1 study testing Baricitinib in people with NMO Spectrum Disorder, run by Tianjin Medical University General Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- NMO Spectrum Disorder
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteChina
- Study dates
- 15 Apr 2023to 1 Mar 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 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;
- Expanded disability status scale (EDSS) score ≤ 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.
Who cannot join
- 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 study within the last 3 months;
- Tumor disease currently or within the last 5 years;
- Pregnancy, breastfeeding, or child-bearing potential during the course of the study;
- Patients with clinically relevant heart, liver, kidney or bone marrow dysfunction;
- History of venous thromboembolism (VTE), or are considered at high risk for VTE by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, 300052
About this study
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.
Read the 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.
- Conditions
- NMO Spectrum Disorder
- Being tested
- Baricitinib
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaricitinibDrug
Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96.
| Study group | Role | What they receive |
|---|---|---|
| Baricitinib | Experimental | Baricitinib will be taken orally with a dose of 4mg once daily until the disease relapses or week 96. Receives: Drug: Baricitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline to 96 weeks
Secondary outcomes (5)
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.
Measured: 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
Measured: 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
Measured: From baseline to 96 weeks
Changes in serum AQP4-IgG titer
Compare serum AQP4-IgG titers before and two year after initial intervention
Measured: From baseline to 96 weeks
Incidence of treatment-emergent adverse events [safety and tolerability]
Adverse events related to baricitinib are recorded
Measured: From baseline to 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2023
- Study start15 Apr 2023
- Primary completion1 Mar 2027 (estimated)
- Study completion1 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NMO Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Tang-Du Hospital, The Second Hospital of Shandong University
- Responsible party
- Principal investigator, Qiang Liu, Tianjin Medical University General Hospital
- Investigators
- Qiang Liu, M.D.,PhD, Principal investigator, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05792462
- Sponsor's protocol ID
- IRB2023-YX-012-01
- Status last verified
- Oct 2025
- First submitted
- 18 Mar 2023
- Last updated
- 11 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No