Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Study Overview
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Brief Summary
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Neuromyelitis Optica Spectrum Disorders (NMOSD) is associated with a pathological humoral immune response against the aquaporin-4(AQP-4) water channel. Rucotinib is an oral inhibitor of JAK1 and JAK2 tyrosine kinases. It may benefit some patients with NMOSD due to the important role of JAK/STAT signaling pathway in the pathogenesis of NMOSD. Clincial trials may be needed to observe its efficacy and safety.

Official Title
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Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Conditions
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Neuromyelitis Optica Spectrum Disorder Relapse

Intervention / Treatment
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* Drug: Ruxolitinib


Participation Criteria
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Eligibility Criteria
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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 at least 2 relapses in last 12 months or 3 relapses in the last 24 months; EDSS <= 6.0; Rituximab should be used for at least 3 months if the condition is stable; 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; Patients taking oral immunosuppressants such as azathioprine; Tumor disease currently or within last 5 years; Pregnant, breastfeeding, or child-bearing potential during the course of the study; Clinically relevant anemia, thrombocytopenia and dysfunction of the heart, liver, kidney or bone marrow.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ruxolitinib<br>Treatment with ruxolitinib will be initiated in an initial dose regimen of 5-10 mg twice daily. Two months later, the dose of ruxolitinib will be increased to 10-15 mg twice daily. | Drug: Ruxolitinib<br>* Treatment with ruxolitinib will be initiated in an initial dose regimen of 5-10 mg twice daily. Two months later, the dose of ruxolitinib will be increased to 10-15 mg twice daily.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| time to the first protocol-defined 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 |
| --- | --- | --- |
| Worsening 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. | Worsening from baseline in EDSS to 52 weeks | 
| Incidence of treatment-emergent adverse events [safety and tolerability] | Adverse events related to ruxolitinib are recorded | From baseline to 52 weeks |