Neuromyelitis Optica Spectrum Disorder Relapse
Conditions
Brief summary
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.
Interventions
DRUGRuxolitinib
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.
Sponsors
Tianjin Medical University General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| time to the first protocol-defined 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Incidence of treatment-emergent adverse events [safety and tolerability] | From baseline to 52 weeks | Adverse events related to ruxolitinib are recorded |
Other
| Measure | Time frame | Description |
|---|---|---|
| Counts of peripheral blood B cell subsets | From baseline to 52 weeks | Compare peripheral blood plasma cells before and one year after initial intervention. |
| 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. |
| Determination of serum AQP4 antibodies | From baseline to 52 weeks | Compare serum AQP4-ab titers before and one year after initial intervention. |
Countries
China
Outcome results
None listed