Inhaled gene therapy aims to fix cystic fibrosis lungs
NCT ID NCT05248230
First seen Jun 25, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This phase 1/2 trial tests a single inhaled dose of gene therapy called 4D-710 in 30 adults with cystic fibrosis who cannot take standard modulator drugs. The therapy delivers a working copy of the CFTR gene directly to lung cells. The main goal is to check safety and side effects, but researchers will also look for signs that the treatment is improving lung function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- 4D-710 (gene therapy delivered via inhalation)
- What this could lead to
- If successful, this could provide a new treatment option for cystic fibrosis lung disease, especially for people who cannot take current modulator therapies.
- What could go wrong
- This is an early phase trial with only 30 participants, so safety and effectiveness are not yet proven. Gene therapy may not reach enough lung cells or could cause side effects.
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
Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Johns Hopkins Hospital
RECRUITINGBaltimore, Maryland, 21287, United States
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Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29425, United States
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National Jewish Health
RECRUITINGDenver, Colorado, 80206, United States
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Northwestern Memorial Hospital
RECRUITINGChicago, Illinois, 60611, United States
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Penn State Health
WITHDRAWNHershey, Pennsylvania, 17033, United States
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Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
RECRUITINGCleveland, Ohio, 44146, United States
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The Hospital of the University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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The University of Arizona
RECRUITINGTucson, Arizona, 85724, United States
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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University of Alabama Child Health Research Unit
RECRUITINGBirmingham, Alabama, 35233, United States
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University of California San Francisco
RECRUITINGSan Francisco, California, 94143, United States
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Miami Hospital
RECRUITINGMiami, Florida, 33136, United States
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University of North Carolina at Chapel Hill
COMPLETEDChapel Hill, North Carolina, 27599, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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Virginia Commonwealth University Health System
RECRUITINGRichmond, Virginia, 23298, United States
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