Skip to main content
TTrialFinder
TrialFinder is for informational purposes only and does not provide medical advice. Always talk to your doctor.
RECRUITINGOBSERVATIONAL

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)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

Who May Be Eligible (Plain English)

Who May Qualify: - 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. Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators

Locations (20)

The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, United States
Stanford University Medical Center
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Clinic
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
The Nemours Children's Clinic - Orlando
Orlando, Florida, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, United States
Children's Mercy Kansas City
Kansas City, Missouri, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
The Cystic Fibrosis Center of Western New York
Buffalo, New York, United States
Children's Hospital of New York
New York, New York, United States
SUNY Upstate Medical University
Syracuse, New York, United States
New York Medical College at Westchester Medical Center
Valhalla, New York, United States