Early use of powerful CF drugs may boost growth in young children
NCT ID NCT04509050
First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time
Summary
This study looks at how highly effective CFTR modulators—medicines like Kalydeco and Trikafta—affect growth and health in infants and young children with cystic fibrosis. Researchers will track changes in weight and height over time in children under 10 years old. The goal is to understand whether starting these drugs early can improve long-term outcomes.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ivacaftor (Kalydeco) or elexacaftor/tezacaftor/ivacaftor (Trikafta)
- What this could lead to
- If successful, this could show that early, highly effective treatment improves growth and long-term health in children with cystic fibrosis.
- What could go wrong
- This is an observational study, not a controlled trial, so results may not prove cause and effect. Side effects of the drugs are possible, and benefits may vary.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Baylor College of Medicine
RECRUITINGHouston, Texas, 77030, United States
Contact Email: •••••@•••••
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Email: •••••@•••••
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Children's Hospital of New York
RECRUITINGNew York, New York, 10032, United States
Contact Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
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Children's Hospital of Pittsburgh of UPMC
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Email: •••••@•••••
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Children's Hospitals and Clinics of Minnesota
RECRUITINGMinneapolis, Minnesota, 55404, United States
Contact Email: •••••@•••••
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Children's Mercy Kansas City
RECRUITINGKansas City, Missouri, 64108, United States
Contact Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Email: •••••@•••••
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Cook Children's Medical Center
RECRUITINGFort Worth, Texas, 76104, United States
Contact Email: •••••@•••••
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Helen DeVos Children's Hospital
RECRUITINGGrand Rapids, Michigan, 49503, United States
Contact Email: •••••@•••••
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Hershey Medical Center Pennsylvania State University
RECRUITINGHershey, Pennsylvania, 17033, United States
Contact Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
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Nemours Children's Clinic
RECRUITINGJacksonville, Florida, 32207, United States
Contact Email: •••••@•••••
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New York Medical College at Westchester Medical Center
RECRUITINGValhalla, New York, 10595, United States
Contact Email: •••••@•••••
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Oklahoma Cystic Fibrosis Center
RECRUITINGOklahoma City, Oklahoma, 73104, United States
Contact Email: •••••@•••••
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Oregon Health Sciences University
RECRUITINGPortland, Oregon, 97239, United States
Contact Email: •••••@•••••
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Primary Children's Cystic Fibrosis Center
RECRUITINGSalt Lake City, Utah, 84113, United States
Contact Email: •••••@•••••
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Riley Hospital for Children
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact Email: •••••@•••••
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SUNY Upstate Medical University
RECRUITINGSyracuse, New York, 13210, United States
Contact Email: •••••@•••••
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
Contact Email: •••••@•••••
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St. Louis Children's Hospital
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Email: •••••@•••••
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Stanford University Medical Center
RECRUITINGPalo Alto, California, 94304, United States
Contact Email: •••••@•••••
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The Children's Hospital Alabama, University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
Contact Email: •••••@•••••
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The Cystic Fibrosis Center of Western New York
RECRUITINGBuffalo, New York, 14203, United States
Contact Email: •••••@•••••
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The Minnesota Cystic Fibrosis Center
RECRUITINGMinneapolis, Minnesota, 55455, United States
Contact Email: •••••@•••••
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The Nemours Children's Clinic - Orlando
RECRUITINGOrlando, Florida, 32827, United States
Contact Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Email: •••••@•••••
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
Contact Email: •••••@•••••
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University of Miami
RECRUITINGMiami, Florida, 33136, United States
Contact Email: •••••@•••••
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University of Michigan, Michigan Medicine
RECRUITINGAnn Arbor, Michigan, 48109, United States
Contact Email: •••••@•••••
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University of Texas Southwestern / Children's Health
RECRUITINGDallas, Texas, 75207, United States
Contact Email: •••••@•••••
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University of Virginia
RECRUITINGCharlottesville, Virginia, 22903, United States
Contact Email: •••••@•••••
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University of Wisconsin
RECRUITINGMadison, Wisconsin, 53792, United States
Contact Email: •••••@•••••
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Vermont Children's Hospital
RECRUITINGBurlington, Vermont, 05401, United States
Contact Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a peer coach help young people take control of their chronic illness?
- Could a single antibiotic be enough for cystic fibrosis lung infections?
- Hands-On therapy may soothe cystic fibrosis pain without drugs
- Newborn screening study aims to catch rare diseases at birth
- Breath sniffing may reveal early clues to cystic fibrosis drug success
- Could a common laxative soothe gut inflammation in cystic fibrosis?