RecruitingNot Applicable

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

United States210 participantsStarted 2020-11-18

Plain-language summary

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 can participate

Age range

10 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part A Primary Outcome Measure: Change in weight-for-age z-scores

Timeframe: Baseline to 12 months

Part A Primary Outcome Measure: Change in height-for-age z-scores

Timeframe: Baseline to 12 months

Part B Primary Outcome Measure: Change in weight-for-age z-scores

Timeframe: Baseline to 12 months

Part B Primary Outcome Measure: Change in height-for-age z-scores

Timeframe: Baseline to 12 months

Trial details

NCT IDNCT04509050

SponsorSonya Heltshe

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-12-31

Contact for this trial

Rachael Buckingham

206-884-7517 rachael.buckingham@seattlechildrens.org

View on ClinicalTrials.gov More Cystic Fibrosis trials

Who can participate

Age range

10 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Part A Primary Outcome Measure: Change in weight-for-age z-scores

Timeframe: Baseline to 12 months

Part A Primary Outcome Measure: Change in height-for-age z-scores

Timeframe: Baseline to 12 months

Part B Primary Outcome Measure: Change in weight-for-age z-scores

Timeframe: Baseline to 12 months

Part B Primary Outcome Measure: Change in height-for-age z-scores

Timeframe: Baseline to 12 months