JAK Inhibitor Treatment in AGS

Conditions

• Aicardi Goutieres Syndrome

Eligibility Criteria

Sex

ALL

Age

1 Month - N/A

Healthy Volunteers

Not accepted

Interventions

• Baricitinib — DRUG
  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.

Study Details

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

Key Dates

Start date

Jun 3, 2019

Status verified

Dec 2025

Primary completion

Jan 4, 2024

Completion

Mar 25, 2024

Study Design

Enrollment

54 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Aicardi Goutières Syndrome patients receiving 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.

Primary Outcome Measure

Mean and Standard Deviation (SD) of the AGS Scale at 52 Weeks [ Time Frame: 52 weeks ]

Locations (1)

Find similar trials in Philadelphia, PA

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