Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Neuromyelitis Optica Spectrum Disorder Relapse Clinical Trial

Official title:

Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT05909943
• Other study ID #: IRB2022-YX-221-01
• Status: Withdrawn
• Phase: Phase 1/Phase 2
• Start date: June 1, 2024
• Est. completion date: August 1, 2026

Study information

• Verified date: June 2023
• Source: Tianjin Medical University General Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: August 1, 2026
• Est. primary completion date: February 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder Relapse

Intervention

Drug:
• Ruxolitinib
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.

Locations

Country	Name	City	State
China	Tianjin Medical University General Hospital	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Tianjin Medical University General Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Counts of peripheral blood B cell subsets	Compare peripheral blood plasma cells before and one year after initial intervention.	From baseline to 52 weeks
Other	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.	From baseline to 52 weeks
Other	Determination of serum AQP4 antibodies	Compare serum AQP4-ab titers before and one year after initial intervention.	From baseline to 52 weeks
Primary	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	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
Secondary	Incidence of treatment-emergent adverse events [safety and tolerability]	Adverse events related to ruxolitinib are recorded	From baseline to 52 weeks