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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)

Who can join

Up to age 10 · All sexes

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.

About the 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).

What is being tested

• Ivacaftor or elexacaftor/tezacaftor/ivacaftor (drug)

Sponsor: Sonya Heltshe · Participants: 210 · Started: Nov 18, 2020

Contact the study team

• Rachael Buckingham · Phone: 206-884-7517
• Anna Mead · Phone: 206-884-7531

Official record on ClinicalTrials.gov — NCT04509050

Locations in the U.S.

Conditions

• Cystic Fibrosis

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.