U54AI198184

Pediatric fungal network study of rare invasive fungal diseases in immunocompromised pediatric patients (PFN-STRIDE) - 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.

The Children's Hospital Of Philadelphia

TO ASSIST PUBLIC AND PRIVATE NONPROFIT INSTITUTIONS AND INDIVIDUALS TO ESTABLISH, EXPAND AND IMPROVE BIOMEDICAL RESEARCH AND RESEARCH TRAINING IN INFECTIOUS DISEASES AND RELATED AREAS; TO CONDUCT DEVELOPMENTAL RESEARCH, TO PRODUCE AND TEST RESEARCH MATERIALS. TO ASSIST PUBLIC, PRIVATE AND COMMERCIAL INSTITUTIONS TO CONDUCT DEVELOPMENTAL RESEARCH, TO PRODUCE AND TEST RESEARCH MATERIALS, TO PROVIDE RESEARCH SERVICES AS REQUIRED BY THE AGENCY FOR PROGRAMS IN INFECTIOUS DISEASES, AND CONTROLLING DISEASE CAUSED BY INFECTIOUS OR PARASITIC AGENTS, ALLERGIC AND IMMUNOLOGIC DISEASES AND RELATED AREAS. PROJECTS RANGE FROM STUDIES OF MICROBIAL PHYSIOLOGY AND ANTIGENIC STRUCTURE TO COLLABORATIVE TRIALS OF EXPERIMENTAL DRUGS AND VACCINES, MECHANISMS OF RESISTANCE TO ANTIBIOTICS AS WELL AS RESEARCH DEALING WITH EPIDEMIOLOGICAL OBSERVATIONS IN HOSPITALIZED PATIENTS OR COMMUNITY POPULATIONS AND PROGRESS IN ALLERGIC AND IMMUNOLOGIC DISEASES. BECAUSE OF THIS DUAL FOCUS, THE PROGRAM ENCOMPASSES BOTH BASIC RESEARCH AND CLINICAL RESEARCH. SMALL BUSINESS INNOVATION RESEARCH (SBIR) PROGRAM EXPANDS AND IMPROVES PRIVATE SECTOR PARTICIPATION IN BIOMEDICAL RESEARCH. THE SBIR PROGRAM INTENDS TO INCREASE AND FACILITATE PRIVATE SECTOR COMMERCIALIZATION OF INNOVATIONS DERIVED FROM FEDERAL RESEARCH AND DEVELOPMENT; TO INCREASE SMALL BUSINESS PARTICIPATION IN FEDERAL RESEARCH AND DEVELOPMENT; AND TO FOSTER AND ENCOURAGE PARTICIPATION OF SOCIALLY AND ECONOMICALLY DISADVANTAGED SMALL BUSINESS CONCERNS AND WOMEN-OWNED SMALL BUSINESS CONCERNS IN TECHNOLOGICAL INNOVATION. THE SMALL BUSINESS TECHNOLOGY TRANSFER (STTR) PROGRAM STIMULATES AND FOSTERS SCIENTIFIC AND TECHNOLOGICAL INNOVATION THROUGH COOPERATIVE RESEARCH AND DEVELOPMENT CARRIED OUT BETWEEN SMALL BUSINESS CONCERNS AND RESEARCH INSTITUTIONS; TO FOSTER TECHNOLOGY TRANSFER BETWEEN SMALL BUSINESS CONCERNS AND RESEARCH INSTITUTIONS; TO INCREASE PRIVATE SECTOR COMMERCIALIZATION OF INNOVATIONS DERIVED FROM FEDERAL RESEARCH AND DEVELOPMENT; AND TO FOSTER AND ENCOURAGE PARTICIPATION OF SOCIALLY AND ECONOMICALLY DISADVANTAGED SMALL BUSINESS CONCERNS AND WOMEN-OWNED SMALL BUSINESS CONCERNS IN TECHNOLOGICAL INNOVATION. RESEARCH CAREER DEVELOPMENT AWARDS SUPPORT THE DEVELOPMENT OF SCIENTISTS DURING THE FORMATIVE STAGES OF THEIR CAREERS. INDIVIDUAL NATIONAL RESEARCH SERVICE AWARDS (NRSAS) ARE MADE DIRECTLY TO APPROVE APPLICANTS FOR RESEARCH TRAINING IN SPECIFIED BIOMEDICAL SHORTAGE AREAS. IN ADDITION, INSTITUTIONAL NATIONAL RESEARCH SERVICE AWARDS ARE MADE TO ENABLE INSTITUTIONS TO SELECT AND MAKE AWARDS TO INDIVIDUALS TO RECEIVE TRAINING UNDER THE AEGIS OF THEIR INSTITUTIONAL PROGRAM.

93.865 - Child Health and Human Development Extramural Research

National Institute of Allergy and Infectious Diseases (NIAID) [HHS - NIH]

National Institute of Child Health and Human Development (NICHD) [HHS - NIH]

Pennsylvania United States

State-Wide

Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional) (PAR-24-206)

Amendment Since initial award the total obligations have increased 369% from $411,231 to $1,927,578.