Studies how cystic fibrosis medicines affect infants and young children
Follows young children with cystic fibrosis taking CFTR modulator medicines over time.
Plain-language title generated from the registry record
Registry title: Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Key facts
- Where
- Ann Arbor, Aurora, Birmingham, Boston, Buffalo, Burlington, Charlottesville, Cincinnati, Columbus, Dallas, Fort Worth, Grand Rapids, Hershey, Houston, Indianapolis, Iowa City, Jacksonville, Kansas City, Madison, Miami, Minneapolis, New York, Oklahoma City, Orlando, Palo Alto, Philadelphia, Pittsburgh, Portland, Salt Lake City, Seattle, St Louis, Syracuse, and Valhalla, United StatesSee the 35 sites
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for infants and children with cystic fibrosis. It studies the effects of highly effective CFTR modulator medicines. It has two parts, A and B.
- Age
- Part A: younger than 10 years. Part B: took part in Part A, or younger than 7 years.
- Sex
- All sexes can join.
- Disease stage
- A documented diagnosis of cystic fibrosis.
- Earlier treatment
- Cannot have used ivacaftor or elexacaftor/tezacaftor/ivacaftor in the 28 days before the first study visit.
- Biomarker or test result
- Part B: CFTR mutations that fit the FDA labeled use of ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Main reasons for not taking part
- Used an investigational drug within 28 days before the first study visit
- Used ivacaftor or elexacaftor/tezacaftor/ivacaftor within 28 days before the first study visit
- Used long-term oral corticosteroids within 28 days before the first study visit
Read by machine on 9 November 2026.
Where in United States
- University of Michigan - Michigan MedicineAnn Arbor, United StatesTaking people
- Children's Hospital ColoradoAurora, United StatesTaking people
- The Children's Hospital Alabama & University of Alabama at BirminghamBirmingham, United StatesTaking people
- Boston Children's HospitalBoston, United StatesTaking people
- The Cystic Fibrosis Center of Western New YorkBuffalo, United StatesTaking people
- Vermont Children's HospitalBurlington, United StatesTaking people
- University of VirginiaCharlottesville, United StatesTaking people
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- University of Texas Southwestern, Children's HealthDallas, United StatesTaking people
- Cook Children's Medical CenterFort Worth, United StatesTaking people
- Helen DeVos Children's HospitalGrand Rapids, United StatesTaking people
- Hershey Medical Center Pennsylvania State UniversityHershey, United StatesTaking people
- Baylor College of MedicineHouston, United StatesTaking people
- Riley Hospital for ChildrenIndianapolis, United StatesTaking people
- University of IowaIowa City, United StatesTaking people
- Nemours Children's ClinicJacksonville, United StatesTaking people
- Children's Mercy Kansas CityKansas City, United StatesTaking people
- University of Kansas Medical CenterKansas City, United StatesTaking people
- University of WisconsinMadison, United StatesTaking people
- University of MiamiMiami, United StatesTaking people
- Children's Hospitals and Clinics of MinnesotaMinneapolis, United StatesTaking people
- The Minnesota Cystic Fibrosis CenterMinneapolis, United StatesTaking people
- Children's Hospital of New YorkNew York, United StatesTaking people
- Oklahoma Cystic Fibrosis CenterOklahoma City, United StatesTaking people
- The Nemours Children's Clinic - OrlandoOrlando, United StatesTaking people
- Stanford University Medical CenterPalo Alto, United StatesTaking people
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
- Children's Hospital of Pittsburgh of UPMCPittsburgh, United StatesTaking people
- Oregon Health Sciences UniversityPortland, United StatesTaking people
- Primary Children's Cystic Fibrosis CenterSalt Lake City, United StatesTaking people
- Seattle Children's HospitalSeattle, United StatesTaking people
- St. Louis Children's HospitalSt Louis, United StatesTaking people
- SUNY Upstate Medical UniversitySyracuse, United StatesTaking people
- New York Medical College at Westchester Medical CenterValhalla, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Sonya Heltshe
- Trial code
- NCT04509050
- Record updated
- 8 July 2026
- Original 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 on children with cystic fibrosis (CF).Read full original descriptionShow less
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
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).
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: 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; Part A and Part B:.