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Clinical Trials in the UK / NCT05668741
Active, not recruiting Phase 1/2

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

NCT05668741 · tracked via the Priya Life Science UK tracker
Sponsor
Vertex Pharmaceuticals Incorporated
Phase
Phase 1/2
Started
2023-02-27
Last updated
2026-09-08

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

VX-522 mRNA therapyIVA

VX-522 mRNA therapy: Oral inhalation using nebulizer.

IVA: Tablet for oral administration.

Study summary

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
University of Alabama at Birmingham - Child Health Research Unit Birmingham Alabama
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach Long Beach California
Stanford University - Palo Alto - Pulmonology Palo Alto California
National Jewish Health Denver Colorado
Clinical & Translational Science Unit (CTSU) - Pulmonology Kansas City Kansas
PAREXEL International - Baltimore Baltimore Maryland
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology Baltimore Maryland
MGH - MGfC Pediatric Cystic Fibrosis Center Boston Massachusetts
Boston Children's Hospital Boston Massachusetts
University of Minnesota -Pulmonology Minneapolis Minnesota
St. Louis Children's Hospital - Pulmonology St Louis Missouri
UC Health Holmes Hospital Cincinnati Ohio
Medical University of South Carolina - Pulmonology Charleston South Carolina
University of Utah Hospital - Pulmonology Salt Lake City Utah
The Alfred Hospital - Pulmonology Melbourne Australia
Universitair Ziekenhuis Gent Ghent Belgium
University of Calgary Medical Clinic of the Foothills Medical Centre Calgary Canada
IUCPQ Pavillon Recherche U-1771 Québec Canada
Ruhrlandklinik Essen Germany
Karolinska University Hospital - Pulmonology Stockholm Sweden
Papworth Hospital NHS Foundation Trust Cambridge United Kingdom
Queen Elizabeth University Hospital - Pulmonology Glasgow United Kingdom
Royal Brompton Hospital London United Kingdom
Wythenshawe Hospital - OPD Manchester United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough Penarth United Kingdom
University Hospital Southampton NHS Fountion - Southampton General Hospital Southampton United Kingdom

More Vertex Pharmaceuticals Incorporated trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05668741 on ClinicalTrials.gov ↗ ← All trials in the UK