PKDNet: a learning network for people with ARPKD
PKDNet - ARPKD Learning Network
['FUNDING_R01'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-11193463
Building a national network and data resource to speed safe clinical trials for children and young adults with autosomal recessive polycystic kidney disease (ARPKD).
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11193463 on ClinicalTrials.gov |
What this research studies
This project will create PKDNet, a coordinated, research-ready cohort of children and young adults with ARPKD by collecting clinical data, imaging, and biosamples across sites. The team will standardize how disease outcomes are measured and develop biomarkers that predict which patients are most likely to get worse. PKDNet will also streamline patient recruitment and data sharing so eligible people can be matched to future clinical trials more quickly.
Who could benefit from this research
Good fit: Children, adolescents, and young adults with a confirmed clinical or genetic diagnosis of ARPKD are ideal candidates to join the network.
Not a fit: People without ARPKD, those with other forms of kidney disease, or patients who are too ill to participate may not be eligible or directly benefit from this project.
Why it matters
Potential benefit: If successful, PKDNet could make it faster to find and test treatments that slow kidney and liver damage in ARPKD.
How similar studies have performed: Animal studies and one small Phase 1 trial have suggested possible therapies, but large human natural history studies and trials are still limited, making this network a novel effort.
Where this research is happening
PHILADELPHIA, UNITED STATES
- 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 below.