New mRNA inhalation therapy offers hope for Hard-to-Treat cystic fibrosis
NCT ID NCT05668741
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 40 times
Summary
This early-stage trial tests an inhaled mRNA therapy called VX-522, combined with an existing drug (ivacaftor), in 26 adults with cystic fibrosis who have genetic mutations that do not respond to current modulator treatments. The main goals are to check safety and tolerability, and to see if it can improve lung function. Participants must have stable disease and specific CFTR gene mutations.
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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.
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
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Locations
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All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, United Kingdom
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, Kansas, 66160, United States
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IUCPQ Pavillon Recherche U-1771
Québec, Canada
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Karolinska University Hospital - Pulmonology
Stockholm, Sweden
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MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, Massachusetts, 02114, United States
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Medical University of South Carolina - Pulmonology
Charleston, South Carolina, 29425, United States
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Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, California, 90806, United States
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National Jewish Health
Denver, Colorado, 80206, United States
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PAREXEL International - Baltimore
Baltimore, Maryland, 21225, United States
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Papworth Hospital NHS Foundation Trust
Cambridge, United Kingdom
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Queen Elizabeth University Hospital - Pulmonology
Glasgow, United Kingdom
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Royal Brompton Hospital
London, United Kingdom
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Ruhrlandklinik
Essen, Germany
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St. Louis Children's Hospital - Pulmonology
St Louis, Missouri, 63110, United States
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Stanford University - Palo Alto - Pulmonology
Palo Alto, California, 94304, United States
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The Alfred Hospital - Pulmonology
Melbourne, Australia
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The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, Maryland, 21287, United States
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UC Health Holmes Hospital
Cincinnati, Ohio, 45220, United States
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Universitair Ziekenhuis Gent
Ghent, Belgium
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University Hospital Southampton NHS Fountion - Southampton General Hospital
Southampton, United Kingdom
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University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, 35233, United States
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University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary, Canada
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University of Minnesota -Pulmonology
Minneapolis, Minnesota, 55455, United States
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University of Utah Hospital - Pulmonology
Salt Lake City, Utah, 84132, United States
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Wythenshawe Hospital - OPD
Manchester, United Kingdom
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
VX-522 mRNA therapy (inhaled) plus ivacaftor tablet
What this could lead to
If successful, this could provide a new treatment option for people with cystic fibrosis who cannot use current modulator therapies, potentially improving lung function and quality of life.
What could go wrong
This is an early-phase trial with only 26 participants, so safety and effectiveness are not yet proven. The therapy is inhaled, which may cause side effects, and it requires ongoing use of ivacaftor.
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.