A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)
J
James Chmiel
Primary Investigator
Recruiting
5 years or below
All
Phase
N/A
2 Locations
Brief description of study
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: cystic fibrosis, Riley
-
Age: 5 years or below
-
Gender: All
Part A of this study will enroll 90 children with confirmed diagnosis of CF who are age < 5 years, and who have not used ivacaftor or elex/tez/iva within the past 180 days. Part B will enroll 120 children, about 80 of whom will have participated in Part A, or who are < 6 years of age; subjects must additionally have an intention to initiate modulator therapy with ivacaftor or elex/tez/iva, and must have a CFTR mutation for which its use is indicated.
Updated on
12 Sep 2024.
Study ID: PPUL-SCRI-BEGIN, 2007020054, TX11391
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