Investigating treatment outcomes in Aicardi Goutières Syndrome
: Clinical Outcomes in Aicardi Goutières Syndrome
This study is looking at how well a medication called baricitinib can help people with Aicardi Goutières Syndrome (AGS) by checking their health and brain scans to see if the treatment makes a difference.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-10653890 on NIH RePORTER |
What this research studies
This research focuses on Aicardi Goutières Syndrome (AGS), a rare genetic disorder that leads to excessive production of interferon, affecting various organs including the brain. The study aims to evaluate the effectiveness of Janus Kinase (JAK) inhibitors, specifically baricitinib, in treating AGS by validating clinical outcome assessments and biomarkers. Patients will be monitored through established functional outcome tools and MRI-based metrics to measure disease progression and treatment response. The research involves collaboration with multiple testing centers to ensure comprehensive data collection and analysis.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with Aicardi Goutières Syndrome, particularly those experiencing severe symptoms.
Not a fit: Patients with other neurological disorders or those not diagnosed with Aicardi Goutières Syndrome may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and better management of Aicardi Goutières Syndrome for affected patients.
How similar studies have performed: Previous research has shown promise in using JAK inhibitors for similar conditions, indicating potential success for this approach in AGS.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Vanderver, Adeline Lucie — Children's Hosp of Philadelphia
- Study coordinator: Vanderver, Adeline Lucie
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.