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New study tracks CF Drug's impact on Kids' lungs and lives

NCT ID NCT04613128

First seen Nov 03, 2025 · Last updated Jun 19, 2026 · Updated 40 times

Summary

This study followed 125 children aged 6-11 with cystic fibrosis who were prescribed a triple therapy (elexacaftor, tezacaftor, and ivacaftor). Researchers measured changes in sweat chloride, lung function, and weight over 24 months to see how well the treatment works in real life. The goal is to understand the therapy's benefits and help guide future care.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital, Brigham & Women's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Mercy Kansas City

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • John Hopkins University

    Baltimore, Maryland, 21287, United States

  • Oregon Health Sciences University

    Portland, Oregon, 97239, United States

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44146, United States

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Minnesota Cystic Fibrosis Center

    Minneapolis, Minnesota, 55455, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of Wisconsin

    Madison, Wisconsin, 53792, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23219, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

elexacaftor, tezacaftor, and ivacaftor (triple combination therapy)

What this could lead to

If successful, this could show that the therapy improves lung function and quality of life in children with cystic fibrosis, guiding future treatments.

What could go wrong

This is an observational study, not a controlled trial, so results may be less definitive. The therapy is already FDA-approved for this age group, so the main goal is to understand real-world effects, not test a new cure.

Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.