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

NCT ID NCT05668741

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 09, 2026 · Updated 2 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.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

26 people

The number who actually took part.

Started

Feb 2023

Expected to finish

Sep 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2) * A total body weight greater than (\>) 50 kg * Stable CF disease * CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G-\>T) * Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to (≥)40 percent (%), MAD: ≥ 50% to less than or equal to (≤) 90% Key Exclusion Criteria: * History of uncontrolled asthma within a year prior to screening * History of solid organ or hematological transplantation * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) * Arterial oxygen saturation on room air less than (\<) 94% at screening Other protocol defined Inclusion/Exclusion criteria may apply.

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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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