Promoting bile duct development in Alagille syndrome
Targeting POGLUT1 to promote biliary development in Alagille syndrome
['FUNDING_R01'] · BAYLOR COLLEGE OF MEDICINE · NIH-10887491
This study is looking at a protein called POGLUT1 to see if it can help improve bile duct development in people with Alagille syndrome, with the hope of making their liver function better and easing some of their symptoms.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BAYLOR COLLEGE OF MEDICINE (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-10887491 on ClinicalTrials.gov |
What this research studies
This research focuses on Alagille syndrome, a genetic disorder that severely affects the liver's bile duct system, leading to complications like cholestasis and liver disease. The study aims to target a specific protein, POGLUT1, to enhance the development of bile ducts in affected individuals. By understanding the underlying mechanisms of bile duct formation, the research seeks to develop a potential therapy that could alleviate symptoms and improve liver function. Patients will be monitored for changes in bile duct development and overall liver health as part of the research.
Who could benefit from this research
Good fit: Ideal candidates for this research are children under 11 years old diagnosed with Alagille syndrome.
Not a fit: Patients with Alagille syndrome who are already candidates for liver transplantation or have severe complications from the disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options for patients with Alagille syndrome, potentially reducing the need for liver transplants.
How similar studies have performed: While there has been significant research into the Notch signaling pathway, this specific approach targeting POGLUT1 in Alagille syndrome is novel and has not been extensively tested.
Where this research is happening
HOUSTON, UNITED STATES
- BAYLOR COLLEGE OF MEDICINE — HOUSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: JAFAR-NEJAD, HAMED — BAYLOR COLLEGE OF MEDICINE
- Study coordinator: JAFAR-NEJAD, HAMED
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.
Conditions: Alagille Syndrome, Alagille-Watson Syndrome