A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
Sonya Heltshe
Summary
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Description
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. Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth fac…
Eligibility
- Age range
- Up to 10 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Part A: * Less than 10 years of age at the first study visit. * Documentation of a CF diagnosis. Part B: * Participated in Part A OR less than 7 years of age at the first study visit. * Documentation of a CF diagnosis. * CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor). * Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor. Exclusion Criteria: * Part A and Part B: * Use of an investigational drug within 28 days prior to and including the first study…
Interventions
- DrugIvacaftor or elexacaftor/tezacaftor/ivacaftor
In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
Locations (35)
- The Children's Hospital Alabama, University of Alabama at BirminghamBirmingham, Alabama
- Stanford University Medical CenterPalo Alto, California
- Children's Hospital ColoradoAurora, Colorado
- Nemours Children's ClinicJacksonville, Florida
- University of MiamiMiami, Florida
- The Nemours Children's Clinic - OrlandoOrlando, Florida