Pediatric Fungal Network (PFN) STudy of Rare Invasive Fungal DisEases in Immunocompromised Pediatric Patients (PFN-STRIDE) Pilot/Feasibility Governance Core

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Abstract

PILOT/FEASIBILITY GOVERNANCE CORE ABSTRACT The Pediatric Fungal Network STudy of Rare Invasive Fungal DisEases in Immunocompromised Pediatric Patients (PFN-STRIDE) Rare Diseases Clinical Research Consortium (RDCRC) Pilot/Feasibility Governance Core will aim to increase the generation of high-quality preliminary data on rare pediatric invasive fungal diseases (IFD) by investigators with a broad range of expertise to advance the overarching goals of the PFN-STRIDE. The Pilot/Feasibility Core will accomplish its aims by soliciting new projects and supporting new investigators. Dr. Anna Huppler will oversee these efforts through leadership of the Pilot Program Committee, which will interface directly with the PFN-STRIDE Executive Committee and the PFN-STRIDE multi-PIs. Members of the Patient Advocacy Group, representatives from the other cores, and site PIs will provide input for the selection of pilot topics and awardees. The overall organization and administration of the Pilot/Feasibility Core will ensure integration with all projects and other cores for the PFN-STRIDE. The activities of the Pilot/Feasibility Core will occur in several stages. The first three stages comprise the activities of Aim 1: to solicit, review, and award Pilot/Feasibility projects that address critical gaps in knowledge in pediatric IFD clinical trial readiness. The first stage is consensus building for pilot topics including input from clinicians, researchers, patients and their families and caregivers. The next stage is solicitation and outreach for applications and investigators occurring in parallel with review committee formation with inclusion of junior investigators. A goal of this stage will be the recruitment of a broad range of investigators to support entry into the investigation of rare pediatric IFD. The third stage will be application review and award selection. Aim 2 of the Pilot/Feasibility Core will implement two stages to establish infrastructure to support Pilot/Feasibility award recipients before and during their project period. Aim 2’s first stage will ensure awardees have support for regulatory compliance, facilitate awardee connection to PFN-STRIDE resources, and integrate pilot/feasibility data with PFN-STRIDE data. In the second stage of Aim 2, Milestone-based progress reports will be reviewed to measure output of key preliminary data in pediatric IFD clinical trial readiness. The expected outcome of the Pilot/Feasibility Core is the development of new lines of investigation and investigators to address the needs of immunocompromised pediatric patients experiencing rare IFD.