Status:
RECRUITING
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Lead Sponsor:
Sonya Heltshe
Collaborating Sponsors:
Cystic Fibrosis Foundation
University of Washington
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
Up to 10 years
Brief 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 wit...
Detailed 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 wit...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 18 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2029
Estimated Enrollment :
210 Patients enrolled
Trial Details
Trial ID
NCT04509050
Start Date
November 18 2020
End Date
December 1 2029
Last Update
August 12 2024
Active Locations (35)
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1
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
Stanford University Medical Center
Palo Alto, California, United States, 94304
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207