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JAK Inhibitor Treatment in AGS

NCT03921554 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2025-12-30

Study results available
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Summary

The primary objective of this study is to assess safety as well as efficacy of baricitinib, a Janus Kinase (JAK) inhibitor, in patients with Aicardi Goutières Syndrome (AGS), a multisystem heritable disorder of the innate immunity resulting in excessive interferon production

Conditions

  • Aicardi Goutieres Syndrome

Interventions

DRUG

Baricitinib

Baricitinib will be taken by mouth or via gastrostomy feeding tube or nasogastric tube as directed by the study doctor. Baricitinib will be dosed by patient age, weight range and estimated glomerular filtration rate (eGFR). Dosing formulations in use in this study will include 1 mg and 2 mg tablets and will be used without splitting. Dispersion will be permitted to aid in swallowing.

Sponsors & Collaborators

Principal Investigators

  • Adeline Vanderver, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-03
Primary Completion
2024-01-04
Completion
2024-03-25
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03921554 on ClinicalTrials.gov