New mRNA inhalation therapy offers hope for Hard-to-Treat cystic fibrosis

NCT ID NCT05668741

Ongoing Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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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.

CYSTIC FIBROSIS

Contacts and locations

Locations

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Penarth, United Kingdom
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Clinical & Translational Science Unit (CTSU) - Pulmonology

Kansas City, Kansas, 66160, United States
IUCPQ Pavillon Recherche U-1771

Québec, Canada
Karolinska University Hospital - Pulmonology

Stockholm, Sweden
MGH - MGfC Pediatric Cystic Fibrosis Center

Boston, Massachusetts, 02114, United States
Medical University of South Carolina - Pulmonology

Charleston, South Carolina, 29425, United States
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
National Jewish Health

Denver, Colorado, 80206, United States
PAREXEL International - Baltimore

Baltimore, Maryland, 21225, United States
Papworth Hospital NHS Foundation Trust

Cambridge, United Kingdom
Queen Elizabeth University Hospital - Pulmonology

Glasgow, United Kingdom
Royal Brompton Hospital

London, United Kingdom
Ruhrlandklinik

Essen, Germany
St. Louis Children's Hospital - Pulmonology

St Louis, Missouri, 63110, United States
Stanford University - Palo Alto - Pulmonology

Palo Alto, California, 94304, United States
The Alfred Hospital - Pulmonology

Melbourne, Australia
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology

Baltimore, Maryland, 21287, United States
UC Health Holmes Hospital

Cincinnati, Ohio, 45220, United States
Universitair Ziekenhuis Gent

Ghent, Belgium
University Hospital Southampton NHS Fountion - Southampton General Hospital

Southampton, United Kingdom
University of Alabama at Birmingham - Child Health Research Unit

Birmingham, Alabama, 35233, United States
University of Calgary Medical Clinic of the Foothills Medical Centre

Calgary, Canada
University of Minnesota -Pulmonology

Minneapolis, Minnesota, 55455, United States
University of Utah Hospital - Pulmonology

Salt Lake City, Utah, 84132, United States
Wythenshawe Hospital - OPD

Manchester, United Kingdom

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