Phase
Condition
Lung Disease
Cystic Fibrosis
Scar Tissue
Treatment
IVA
VX-522 mRNA therapy
Clinical Study ID
Ages 18-65 All Genders
Study Summary
Eligibility Criteria
Inclusion
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 modulatortherapy o Example mutations include but are not limited to, mutations that do not produceCFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonicalsplice 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
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 PughScore 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.
Study Design
Connect with a study center
Alfred Hospital
Melbourne,
AustraliaSite Not Available
Universitair Ziekenhuis Gent
Gent,
BelgiumSite Not Available
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary,
CanadaSite Not Available
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
Quebec,
CanadaSite Not Available
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen,
GermanySite Not Available
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova,
ItalySite Not Available
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
Verona,
ItalySite Not Available
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Utrecht,
NetherlandsSite Not Available
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona,
SpainSite Not Available
Hospital Universitario Virgen del Rocio
Sevilla,
SpainSite Not Available
Hospital Universitario y Politecnico La Fe
Valencia,
SpainSite Not Available
Karolinska Universitetssjukhuset, Huddinge
Stockholm,
SwedenSite Not Available
Royal Papworth Hospital NHS Foundation Trust
Cambridge,
United KingdomSite Not Available
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow,
United KingdomActive - Recruiting
Royal Hospital for Children
Glasgow,
United KingdomSite Not Available
Royal Brompton Hospital
London,
United KingdomSite Not Available
Wythenshawe Hospital
Manchester,
United KingdomSite Not Available
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth,
United KingdomSite Not Available
Southampton General Hospital
Southampton,
United KingdomSite Not Available
University of Alabama at Birmingham
Birmingham, Alabama 35233
United StatesSite Not Available
Miller Children's Hospital / Long Beach Memorial
Long Beach, California 90806
United StatesSite Not Available
Stanford University
Palo Alto, California 94304
United StatesSite Not Available
Stanford University Clinical and Translational Research Unit
Palo Alto, California 94304
United StatesSite Not Available
National Jewish Health
Denver, Colorado 80206
United StatesSite Not Available
University of Florida, Shands Hospital
Gainesville, Florida 32610
United StatesSite Not Available
University of Kansas Medical Center
Kansas City, Kansas 66160
United StatesSite Not Available
Baltimore - Early Phase Clinical Unit
Baltimore, Maryland 21225
United StatesSite Not Available
Johns Hopkins Hospital
Baltimore, Maryland 21287
United StatesSite Not Available
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Massachusetts General Hospital
Boston, Massachusetts 02114
United StatesSite Not Available
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
Boston, Massachusetts 02114
United StatesActive - Recruiting
University of Minnesota
Minneapolis, Minnesota 55455
United StatesSite Not Available
Washington University School of Medicine / St. Louis Children's Hospital
Saint Louis, Missouri 63110
United StatesSite Not Available
Columbia University Medical Center
New York, New York 10032
United StatesSite Not Available
Lenox Hill Hospital
New York, New York 10028
United StatesSite Not Available
UC Health Holmes
Cincinnati, Ohio 45220
United StatesSite Not Available
University of Cincinnati
Cincinnati, Ohio 45220
United StatesActive - Recruiting
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106
United StatesSite Not Available
Nationwide Children's Hospital
Columbus, Ohio 43205
United StatesSite Not Available
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
United StatesSite Not Available
Medical University of South Carolina
Charleston, South Carolina 29425
United StatesActive - Recruiting
Medical University of South Carolina - Nexus Research Clinic
Charleston, South Carolina 29425
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesSite Not Available
Baylor College of Medicine
Houston, Texas 77030
United StatesSite Not Available
University of Utah
Salt Lake City, Utah 84132
United StatesSite Not Available
University of Wisconsin Hospital and Clinics
Madison, Wisconsin 53792
United StatesSite Not Available
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