This Project Grant award of $800,000 from the Food and Drug Administration (FDA) Research program (CFDA 93.103) supports a natural history study to advance understanding and treatment of Congenital Central Hypoventilation Syndrome (CCHS), a rare and severe neurocristopathy. The study, led by the Ann & Robert H Lurie Children's Hospital of Chicago, aims to capture longitudinal data on the CCHS patient journey, incorporate the patient voice, harmonize clinical and registry data, and...
This $605,280 federal Project Grant award was provided by the National Institutes of Health (NIH) National Human Genome Research Institute (NHGRI) under the Human Genome Research (CFDA 93.172) program. The objective of this 4-year project is to gain a multi-perspective understanding of the ethical challenges arising in patient-focused drug development (PFDD) for rare diseases and to develop tools to manage these challenges. The project involves three main activities: 1) conducting...
The Yale University Diagnostic Center of Excellence for Undiagnosed Diseases (YDCOE) was awarded a $800,000 Cooperative Agreement under the Trans-NIH Research Support program (CFDA 93.310) funded by the National Institutes of Health (NIH) Office of the Director. The YDCOE project aims to expand access to the Undiagnosed Diseases Network (UDN) by partnering with community healthcare organizations serving underinsured and minority populations. The YDCOE will leverage Yale's expertise in human...
The National Center for Advancing Translational Sciences (NCATS) awarded a $429,000 Project Grant (CFDA 93.350) to The Regents of the University of Colorado (University of Colorado-Denver) to address critical gaps in enabling clinical trial readiness for the rare Posterior Cortical Atrophy (PCA) syndrome. Over a 2-year period from August 2024 to July 2026, the project aims to longitudinally assess patient-reported and informant-reported PCA-specific clinical outcome assessments (COAs),...
The U.S. National Institutes of Health (NIH) awarded a $1,999,508 Cooperative Agreement under the Trans-NIH Research Support program (CFDA 93.310) to RTI International and Emory University to establish the "INCLUDE Down Syndrome Clinical Cohort Coordinating Center (DS-4C)." The DS-4C will serve as the central hub to orchestrate and integrate all components of the INCLUDE project's Down Syndrome Cohort Development Program (DS-CDP), including overseeing the implementation of a common...
This federal Cooperative Agreement award from the Food and Drug Administration (FDA) Research program (CFDA 93.103) provides $1,277,294 to the University of Wisconsin System to enroll 180 individuals with the CADASIL gene mutation in a study investigating retinal imaging measures as potential diagnostic, prognostic, and disease monitoring biomarkers for vascular cognitive impairment and dementia. The study, which began on September 5, 2024, will focus on three primary retinal assessments: 1)...
This federal Cooperative Agreement award from the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA) supports the development of a qualification plan for the Virtual Reality Functional Capacity Assessment Tool-Short List (VRFCAT-SLX) as a performance outcome measure for assessing cognition-dependent daily functioning in people diagnosed with biomarker-positive, clinical stage 2 and 3 Alzheimer's disease (AD). The $249,984 award will fund a project...
This federal Project Grant award of $150,000 from the Food and Drug Administration (FDA) under the FDA Research program (CFDA 93.103) will support the Critical Path Institute's (C-Path) 4-day "C-Path Global Impact Conference." The purpose of this conference is to showcase the work of C-Path's public-private partnerships (PPPs) in generating solutions to accelerate drug development, with a focus on novel therapeutics for challenging conditions in neurology, rare/orphan diseases, and...
This Cooperative Agreement award from the U.S. Food and Drug Administration (FDA) under the FDA Research program (CFDA 93.103) provides $249,826 to The Critical Path Institute (C-Path) to conduct psychometric analyses and prepare a quantitative study report to support the qualification of the Chronic Heart Failure Symptom Scale (CHF-SS) as a clinical outcome assessment tool for use in heart failure drug development. The project aims to finalize the statistical analysis plan, analyze the...
This Cooperative Agreement award from the Food and Drug Administration (FDA) under the FDA Research program (CFDA 93.103) provides $500,000 in funding to the University of Connecticut Health Center (UCHC) to advance linguistic inclusion in clinical trials. The project aims to: Explore Spanish-speaking patients' perspectives, preferences, and barriers regarding participation in clinical trials. Employ a participatory planning process to develop Spanish-language outreach materials, an...