Ireland
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Completed Phase 1/2

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

NCT05668741 · tracked via the Priya Life Science Sweden tracker
Phase
Phase 1/2
Started
2023-02-27
Last updated
2026-10-09

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)

  • Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — From Day 1 Through Week 8 [SAD and MAD]

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
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 Sweden