Core E: Clinical Translation Core

PROJECT SUMMARY/ABSTRACT Improving cystic fibrosis (CF) outcomes have reshaped research priorities in this field. The Clinical Translation Core (CTC) has continued to evolve to meet these research needs. In Aim 1, the CTC provides access to a broad collection of human biospecimens with associated clinical data to investigators conducting basic and translational research. A specific focus on the acquisition of intestinal biopsy samples during clinically indicated endoscopies provides these hard to obtain samples. In Aim 2, the CTC provides access to comprehensive clinical research expertise across gastroenterology, endocrinology, pulmonology, functional lung imaging and biostatistical fields. Importantly, new expertise in diabetes, obesity, and metabolic syndrome has been added to this core. This accumulation of experienced researchers will provide CTC users with content area and study design expertise that is relevant to most major disease manifestations and complications. Further, the embedded biostatistical expertise assists with data analysis, database creation and management, feasibility and sample size estimations. Finally, Aim 3 provides access to specialized pulmonary outcome measures that are well suited to the testing of novel therapeutics, particularly for patients without access to highly effective CFTR modulators, and children with mild disease. Support of these novel outcome measures ensures their ongoing availability to the CF community.