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

Recruiting now

Conditions studied: Cystic Fibrosis

In brief

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).

Key facts

Study ID

NCT04509050

Run by

Sonya Heltshe

People needed

210

Starts

2020-11-18

Expected to finish

2027-12-31

Last updated by the study team

2026-07-08

Who can join

Age: any, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The Children's Hospital Alabama, University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)
• Stanford University Medical Center — Palo Alto, California, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Nemours Children's Clinic — Jacksonville, Florida, United States (enrolling)
• University of Miami — Miami, Florida, United States (enrolling)
• The Nemours Children's Clinic - Orlando — Orlando, Florida, United States (enrolling)
• Riley Hospital for Children — Indianapolis, Indiana, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Michigan, Michigan Medicine — Ann Arbor, Michigan, United States (enrolling)
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States (enrolling)
• Children's Hospitals and Clinics of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• The Minnesota Cystic Fibrosis Center — Minneapolis, Minnesota, United States (enrolling)
• Children's Mercy Kansas City — Kansas City, Missouri, United States (enrolling)
• St. Louis Children's Hospital — St Louis, Missouri, United States (enrolling)
• The Cystic Fibrosis Center of Western New York — Buffalo, New York, United States (enrolling)
• Children's Hospital of New York — New York, New York, United States (enrolling)
• SUNY Upstate Medical University — Syracuse, New York, United States (enrolling)
• New York Medical College at Westchester Medical Center — Valhalla, New York, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
• Oklahoma Cystic Fibrosis Center — Oklahoma City, Oklahoma, United States (enrolling)
• Oregon Health Sciences University — Portland, Oregon, United States (enrolling)
• Hershey Medical Center Pennsylvania State University — Hershey, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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