Cystic Fibrosis CFTR Therapy Outcomes Analysis: The CHEC-SC Cohort Study

Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes

Protocol Description

The purpose of this CHEC-SC study is to test the amount of salt in the sweat of cystic fibrosis patients to find out how different people respond to treatment with CFTR modulator therapy and to see if CFTR therapy impacts long- term health outcomes. Researchers know that some cystic fibrosis patients have different reductions in sweat chloride after treatment with CFTR modulator therapy, but they do not know why or whether the sweat chloride value achieved after such treatment impacts long-term health outcomes.

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to participants of both genders, ages 4 months and older, who have cystic fibrosis and are being treated with a CFTR modulator, which targets the cystic fibrosis transmembrane conductance regulator (CTFR) gene.
Males and females: Ages 4 months and older

Requirements

This study requires a single visit for a physical examination and testing, including spirometry and a sweat chloride test.
Visits: 1
Duration: Not applicable

Status: Open to those enrolled in the Cystic Fibrosis Foundation Patient Registry

Source(s) of Support

Cystic Fibrosis Foundation Therapeutics

Primary Investigator

Joseph Pilewski, MD

Contact Information

For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator
Rose Lanzo BS, RRT, CRC
412-692-5872