Clinical Research Directory

Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)

RECOVER is a prospective, multicenter observational study designed to measure the real world clinical effectiveness of elexacaftor/tezacaftor/ivacaftor (ETI) triple combination therapy in people with cystic fibrosis initially over a two year period, followed by a five year extension period. Measured outcomes include measures of lung function, lung inflammation, lung imaging, abdominal symptoms, gut inflammation, liver function, pancreatic exocrine function, nasal inflammation, quality of life and adherence to therapy. The study will examine outcomes in two cohorts, children aged six to eleven years and children and adults aged twelve and above.

VALidation of Imaging-based Liver Biomarkers in PEDiatric Patients

The goal of this observational (diagnostic validation) study is to validate imaging-based biomarkers for assessing liver involvement in pediatric patients with established or suspected liver disease. The projekt involves 3 larger studies, each with specific aims specified in detailed description below. The main study (study 1) compares imaging-based biomarkers-with liver biopsy to evaluate their correlation with histologically confirmed liver disease, including fibrosis, inflammation, and steatosis. The main question the overall project aims to answer is: • Can multiple novel imaging-based biomarkers, such as shear wave elastography (SWE), shear wave dispersion (SWD), and attenuation imaging (ATI), be used for detailed, non-invasive liver characterization in children, and if so, how should they be used?