Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

NCT05909943 | Withdrawn Phase 1

• 1 other identifier: IRB2022-YX-221-01
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Neuromyelitis Optica Spectrum Disorder Relapse

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Worsening in EDSS

  Worsening from baseline in EDSS to 52 weeks

• Incidence of treatment-emergent adverse events [safety and tolerability]

  From baseline to 52 weeks

Other Outcomes (3)

• Counts of peripheral blood B cell subsets

  From baseline to 52 weeks

• 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

• Determination of serum AQP4 antibodies

  From baseline to 52 weeks

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

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

Interventions

Ruxolitinib DRUG

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.

Ruxolitinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead

Study Sites (1)

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, 300052, China

Location

MeSH Terms

Interventions

ruxolitinib

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology Department

Study Record Dates

• First Submitted: March 8, 2023
• First Posted: June 18, 2023
• Study Start: June 1, 2024
• Primary Completion: February 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: June 18, 2023

Record last verified: 2023-06

Locations

CN (1)