BEGIN-OB-19: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)


This is a study of highly effective CFTR modulators and their impact in children with CF on endocrine growth factors, the gut microbiome, respiratory microbiome, liver and pancreatic function, lung function, sweat chloride, and inflammatory markers.

I'm interested

Male or Female
Up to 18 years old
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• For Part A: less than 5 years of age at the first study visit
• For Part B: participated in Part A OR less than 6 years of age at the first study visit, CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy and physician intends to prescribe ivacaftor or elexacaftor/tezacaftor/ ivacaftor
• Documented diagnosis of Cystic Fibrosis (CF)
Exclusion Criteria:

• use of ivacaftor or elexacaftor/tezacaftor/ ivacaftor within the 180 days
• use of an investigational drug within 28 days prior to first study visit
• use of chronic oral corticosteroids within the 28 days prior to first study visit

Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Rare Diseases

Cystic Fibrosis

Alyssa Perry -
Elissa Downs
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