Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
Sonya Heltshe
210 participants
Nov 18, 2020
OBSERVATIONAL
Conditions
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).
Eligibility
Inclusion Criteria8
- 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 Criteria4
- Part A and Part B:
- 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.
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Interventions
In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
Locations(35)
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NCT04509050