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Recruiting
NCT04509050
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Conditions: Cystic Fibrosis
Sex: All
Ages: N/A – 10 Years
Healthy volunteers: No
Enrollment: 210
Sponsor: Sonya Heltshe
Location: The Children's Hospital Alabama, University of Alabama at Birmingham Birmingham Alabama
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 Criteria
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 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.
Source: ClinicalTrials.gov (NCT04509050). StuddyBuddy aggregates publicly available trial information.