Project Grant R01FD008225

The Children's Hospital of Philadelphia (CHOP) has been awarded a $400,000 Project Grant (R01FD008225) by the Food and Drug Administration (FDA) under the FDA Research program (CFDA 93.103). The objective of this 4-year grant is to accelerate clinical trials for Autosomal Recessive Polycystic Kidney Disease (ARPKD), a rare genetic disorder affecting children and young adults. The grant will fund the creation of PKDNET, a data resource to collect natural history data, identify suitable clinical trial endpoints and prognostic biomarkers, and facilitate future patient recruitment for ARPKD studies. This will be accomplished by leveraging CHOP's participation in the PEDSnet national pediatric learning health system, which provides access to electronic health records for over 13 million children across 11 U.S. academic pediatric centers. The grant aims to address key barriers to ARPKD clinical trials, including limited natural history data and lack of defined outcome measures, to advance the development of potentially disease-modifying therapies for this rare condition.