Creating a network to improve understanding and treatment of ARPKD
Official title: PKDNet - ARPKD Learning Network
Funded at Children's Hosp of Philadelphia · NIH-11034219 · Data updated 2026-11-12
This study is creating a helpful database called PKDnet to find and support young people with autosomal recessive polycystic kidney disease (ARPKD), so we can gather important information and make it easier for them to join future clinical trials that aim to develop better treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 15, 2024 |
| Project end | August 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $400,000 |
| NIH project number | 1R01FD008225-01 |
| Project ID | NIH-11034219 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and young adults diagnosed with autosomal recessive polycystic kidney disease.
✕Less likely to help
Patients with other forms of kidney disease or those who do not have ARPKD may not benefit from this research.
What this research studies
This research focuses on autosomal recessive polycystic kidney disease (ARPKD), a rare condition affecting children and young adults. The project aims to establish PKDnet, a comprehensive data resource that will help identify patients with ARPKD and gather critical information to support clinical trials. By addressing barriers such as limited natural history data and the need for defined clinical outcomes, the research seeks to enhance the recruitment of eligible patients for future studies. Ultimately, this initiative aims to accelerate the development of effective therapies for ARPKD.
Why it matters
Potential benefit: If successful, this research could lead to the development of effective treatments for ARPKD, significantly improving patient outcomes.
How similar studies have gone: While there has been limited research on ARPKD, the establishment of networks for rare diseases has shown promise in facilitating clinical trials in other conditions.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Hartung, Erum Aftab — Children's Hosp of Philadelphia
- Study coordinator: Hartung, Erum Aftab
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.