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Observational Study of CFTR Modulators in Children with Cystic Fibrosis

This study is looking at how well treatments like ivacaftor or elexacaftor/tezacaftor/ivacaftor (CFTR modulators) work in infants and young children with cystic fibrosis (CF). Researchers want to understand how these medicines affect growth, digestion, lung health, and other body functions over time. Children up to 10 years old with a CF diagnosis can join. For some parts of the study, children need to have specific CFTR mutations that respond to these medicines. The main goal is to see changes in weight and height over 12 months. The study status is unclear, and it aims to enroll 210 participants.

Study design
This is an observational study, meaning participants will receive their prescribed CFTR modulator, and researchers will observe its effects. It is a multi-center study with a planned enrollment of 210 participants.
What's involved
In Part A, participants will have up to 8 visits over a period of up to 5 years. In Part B, participants will have 1 visit before starting treatment and 8 visits over a 60-month (5-year) follow-up period.
Compensation
Not stated in the trial record.
Follow-up
Participants in Part B will be followed for 60 months (5 years) after starting treatment.

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NCT04509050

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

Recruiting
Not specifiedUp to 10Observational
Sonya Heltshe
~210 participants
Updated 2026-07-08 on ClinicalTrials.gov
What's tested:Ivacaftor or elexacaftor/tezacaftor/ivacaftor

At a glance

Recruiting sites
35 of 35 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part A Primary Outcome Measure: Change in weight-for-age z-scores
Measured over Baseline to 12 months
+3 more outcomes measured

Conditions

Cystic Fibrosis

Explore related trials

Cystic FibrosisCFTR Gene

Where it's being run

35 sites across 22 states
New York4
Florida3
Pennsylvania3
Texas3
Michigan2
Minnesota2
Missouri2
Ohio2

Study leadership

  • Bonnie Ramsey, MD · PRINCIPAL_INVESTIGATOR · Seattle Children's Hospital
  • Lucas Hoffman, MD PhD · PRINCIPAL_INVESTIGATOR · University of Washington/Seattle Children's
  • Katie Larson Ode, MD · PRINCIPAL_INVESTIGATOR · University of Iowa

Who to contact

Rachael Buckingham
[email protected]
206-884-7517
Email the study team

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Eligibility criteria

Inclusion

Part A:
Less than 10 years of age at the first study visit.
Documentation of a CF diagnosis.
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.

What this trial measures

  • Part A Primary Outcome Measure: Change in weight-for-age z-scoresBaseline to 12 months

    Weight-for-age z-scores over time

  • Part A Primary Outcome Measure: Change in height-for-age z-scoresBaseline to 12 months

    Height-for-age z-scores over time

  • Part B Primary Outcome Measure: Change in weight-for-age z-scoresBaseline to 12 months

    Change in weight-for-age z-scores from baseline

  • Part B Primary Outcome Measure: Change in height-for-age z-scoresBaseline to 12 months

    Change in height-for-age z-scores from baseline