Evaluate the Efficacy of Anti-Jak1 Inhibitors as Treatment for Patients With Aicardi-Goutières Syndrome

Plain-language summary

This study evaluated the efficacy of JAK1 inhibitors (such as Baricitinib or Ruxolitinib) as a treatment for patients with Aicardi-Goutières Syndrome or related interferonopathies. Patients aged 0-25 with a genetic diagnosis of Aicardi-Goutières Syndrome took part. Conducted by IRCCS Fondazione Stella Maris, this study without an assigned phase has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

IRCCS Fondazione Stella Maris

Intervention

JAK Inhibitor

Start date

4/7/2024

Estimated completion

12/18/2024

Countries

Italy

Related genes

Locations

• Pisa, Italy

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* Patients over the age of 25 years
* Patients who have not been genetically diagnosed with Aicardi-Goutières Syndrome (AGS) or AGS-related interferonopathies.