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).
Less than 10 years of age at the first study visit.
Documentation of a CF diagnosis.
Participated in Part A OR less than 7 years of age at the first study visit.
Documentation of a CF diagnosis.
Use of an investigational drug within 28 days prior to and including the first study visit.
Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit.
Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy Part B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy
Cohort
Prospective
Ivacaftor or elexacaftor/tezacaftor/ivacaftor
DrugIn Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
Part A Primary Outcome Measure: Change in weight-for-age z-scores
Weight-for-age z-scores over time
Part A Primary Outcome Measure: Change in height-for-age z-scores
Height-for-age z-scores over time
Part B Primary Outcome Measure: Change in weight-for-age z-scores
Change in weight-for-age z-scores from baseline
Part B Primary Outcome Measure: Change in height-for-age z-scores
Change in height-for-age z-scores from baseline
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Sonya Heltshe
Lead sponsor
Seattle Children's Hospital
Sponsor institution
Cystic Fibrosis Foundation
Collaborator
University of Washington
Collaborator
University of Alabama at Birmingham
Collaborator
