Clinical Trial: A Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders

U.S., Oct. 23 -- ClinicalTrials.gov registry received information related to the study (NCT07889973) titled 'A Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders' on Oct. 12.

Brief Summary: This multicenter translational research study integrates diagnostic analyses and ex-vivo functional testing to support future clinical trials evaluating the safety and efficacy of CFTR modulator treatments in individuals with cystic fibrosis (CF) carrying rare or orphan mutations that may benefit from these therapies.

Patient-derived nasal epithelial cells will be collected through standardized nasal brushing procedures, expanded, and cultured under air-li...