Status:
COMPLETED
Evaluate the Efficacy of Anti-Jak1 Inhibitors as Treatment for Patients With Aicardi-Goutières Syndrome
Lead Sponsor:
IRCCS Fondazione Stella Maris
Conditions:
Aicardi-Goutières Syndrome (AGS)
Eligibility:
All Genders
Up to 25 years
Brief Summary
Aicardi-Goutières Syndrome (AGS) is a hereditary multisystem autoinflammatory disorder that predominantly affects the central nervous system. It is characterized by severe neurological disability and ...
Eligibility Criteria
Inclusion
- Patients aged 0 to 25 years
- Patients who have been genetically diagnosed with Aicardi-Goutières Syndrome (AGS) or AGS-related interferonopathies For case cohort-Patients treated with Janus Kinase 1/2 (JAK1/2) inhibitors, such as Baricitinib or Ruxolitinib
Exclusion
- Patients over the age of 25 years
- Patients who have not been genetically diagnosed with Aicardi-Goutières Syndrome (AGS) or AGS-related interferonopathies.
Key Trial Info
Start Date :
April 7 2024
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 18 2024
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT06898372
Start Date
April 7 2024
End Date
December 18 2024
Last Update
March 27 2025
Active Locations (1)
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1
IRCCS Stella Maris Foundation
Pisa, Italy, 56128