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โ† Trials
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Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

Recruitingn/aAges any - 10 YearsAllSonya Heltshe
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • Ivacaftor or elexacaftor/tezacaftor/ivacaftor
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Where

35 sites
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, United States
Stanford University Medical Center
Palo Alto, United States
Children's Hospital Colorado
Aurora, United States
Nemours Children's Clinic
Jacksonville, United States
University of Miami
Miami, United States
The Nemours Children's Clinic - Orlando
Orlando, United States
Riley Hospital for Children
Indianapolis, United States
University of Iowa
Iowa City, United States

โ€ฆand 27 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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