NCT02710383

Biomarker for Cystic Fibrosis

Biomarker for Cystic Fibrosis: An International, Multicenter, Observational, Longitudinal Protocol

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 54 (actual)
• Sponsor: CENTOGENE GmbH Rostock · Industry
• Sex: All
• Age: 2 Months – 50 Years
• Healthy volunteers: Not accepted

Summary

International, multicenter, observational, longitudinal study to identify biomarker/s for Cystic fibrosis and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s

Detailed description

Cystic fibrosis (CyFi) is a progressive hereditary disease with the prevalence of 1 in 2500. CyFi is an autosomal recessive disease caused by pathogenic variant/s in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene encoding Cftr protein. CyFi causes chronic respiratory damage. Pulmonary findings occur already in infancy, which raises questions whether obstruction might be congenital. Thick, sticky mucus clogs the airways, reduces muco-ciliary clearance and leads to problems with breathing and recurrent bacterial (Pseudomonas aeruginosa) infections, which causes over time the formation of scar tissue (fibrosis) and cysts in the lungs.There is no cure for CyFi; however, symptomatic treatment can help relieve symptoms. The aim of this study is to identify biomarkers for Cystic fibrosis disease and to explore their clinical robustness, specificity, and long-term variability. An ideal biomarker plays an essential role in the early diagnosis, prediction and therapeutic monitoring of a specific disorder.

Conditions

• Chronic Nasal Congestion
• Lung Infection
• Breathlessness
• Clubbing Toes
• Meconium Ileus
• Failure to Thrive
• Pancreatitis

Timeline

• Start date: 2018-08-20
• Primary completion: 2022-12-31
• Completion: 2022-12-31
• First posted: 2016-03-16
• Last updated: 2023-02-10

Locations

5 sites across 5 countries: Albania, Georgia, India, Pakistan, Sri Lanka