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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IRCCS Stella Maris Foundation

Pisa, Italy, 56128