A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT ID: NCT05668741
Last Updated: 2025-11-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
39 participants
INTERVENTIONAL
2023-02-27
2027-05-12
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Single Ascending Dose (SAD)
Participants grouped into different cohorts will receive a single ascending dose of VX-522.
VX-522 mRNA therapy
Oral inhalation using nebulizer.
Multiple Ascending Dose (MAD) Cohort 1: VX-522
Participants will receive multiple ascending doses of VX-522.
VX-522 mRNA therapy
Oral inhalation using nebulizer.
MAD Cohort 1: VX-522+ IVA
Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
VX-522 mRNA therapy
Oral inhalation using nebulizer.
IVA
Tablet for oral administration.
MAD Cohort 2: VX-522+ IVA
Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
VX-522 mRNA therapy
Oral inhalation using nebulizer.
IVA
Tablet for oral administration.
Interventions
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VX-522 mRNA therapy
Oral inhalation using nebulizer.
IVA
Tablet for oral administration.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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: ≥ 40% to less than or equal to (≤) 90%
Exclusion Criteria
* 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
18 Years
65 Years
ALL
No
Sponsors
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Moderna, Inc
UNKNOWN
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, 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, United States
Stanford University - Palo Alto - Pulmonology
Palo Alto, California, United States
National Jewish Health
Denver, Colorado, United States
Shands Hospital - Pulmonology
Gainesville, Florida, United States
Northwestern Memorial Hospital - Feinberg Pavilion
Chicago, Illinois, United States
Clinical & Translational Science Unit (CTSU) - Pulmonology
Kansas City, Kansas, United States
PAREXEL International - Baltimore
Baltimore, Maryland, United States
The Johns Hopkins University - Johns Hopkins Hospital - Pulmonology
Baltimore, Maryland, United States
MGH - MGfC Pediatric Cystic Fibrosis Center
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Minnesota -Pulmonology
Minneapolis, Minnesota, United States
St. Louis Children's Hospital - Pulmonology
St Louis, Missouri, United States
Medicine at East 85th Street
New York, New York, United States
Presbyterian Hospital - Pulmonology
New York, New York, United States
UC Health Holmes Hospital
Cincinnati, Ohio, United States
UH Cleveland Medical Center - Pulmonology
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Pittsburgh - Pulmonology
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina - Pulmonology
Charleston, South Carolina, United States
Vanderbilt Interventional Pulmonology
Nashville, Tennessee, United States
Texas Children's Hospital - Wallace Tower - Pulmonology
Houston, Texas, United States
University of Utah Hospital - Pulmonology
Salt Lake City, Utah, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States
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
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico - Pulmonology
Genova, , Italy
Centro Ricerche Cliniche di Verona S.r.l.
Verona, , Italy
UMCU - Department of Pulmonology and Tuberculosis
Utrecht, , Netherlands
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, , Spain
Hospital Virgen del Rocio - Pulmonology
Seville, , Spain
Hospital Universitario y Politecnico La Fe - Pulmonology
Valencia, , Spain
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
Countries
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Central Contacts
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Other Identifiers
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2022-000726-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-504786-23-00
Identifier Type: OTHER
Identifier Source: secondary_id
VX21-522-001
Identifier Type: -
Identifier Source: org_study_id