A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: VX-522 mRNA therapyDrug: IVA
- Registration Number
- NCT05668741
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 36
-
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: ≥ 40% to less than or equal to (≤) 90%
Key
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Single Ascending Dose (SAD) VX-522 mRNA therapy Participants grouped into different cohorts will receive a single ascending dose of VX-522. Multiple Ascending Dose (MAD) Arm 1: VX-522 VX-522 mRNA therapy Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1). MAD Arm 2: VX522+ IVA VX-522 mRNA therapy Following run-in period with ivacaftor (IVA), participants grouped into different cohorts will receive multiple ascending doses of VX-522 with IVA in treatment arm (T2). MAD Arm 2: VX522+ IVA IVA Following run-in period with ivacaftor (IVA), participants grouped into different cohorts will receive multiple ascending doses of VX-522 with IVA in treatment arm (T2).
- Primary Outcome Measures
Name Time Method Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 Through Safety Follow-up Visit [up to Week 24 for SAD, and Week 28 for T1 and T2 (MAD)]
- Secondary Outcome Measures
Name Time Method MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline at Day 29
Trial Locations
- Locations (41)
UC Health Holmes
🇺🇸Cincinnati, Ohio, United States
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
🇮🇹Verona, Italy
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
🇳🇱Utrecht, Netherlands
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
🇪🇸Barcelona, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain
Karolinska Universitetssjukhuset, Huddinge
🇸🇪Stockholm, Sweden
Royal Hospital for Children
🇬🇧Glasgow, United Kingdom
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Stanford University Clinical and Translational Research Unit
🇺🇸Palo Alto, California, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Florida, Shands Hospital
🇺🇸Gainesville, Florida, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Baltimore - Early Phase Clinical Unit
🇺🇸Baltimore, Maryland, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Lenox Hill Hospital
🇺🇸New York, New York, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Medical University of South Carolina - Nexus Research Clinic
🇺🇸Charleston, South Carolina, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Alfred Hospital
🇦🇺Melbourne, Australia
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
University of Calgary Medical Clinic of the Foothills Medical Centre
🇨🇦Calgary, Canada
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
🇨🇦Quebec, Canada
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
Royal Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
Royal Brompton Hospital
🇬🇧London, United Kingdom
Wythenshawe Hospital
🇬🇧Manchester, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
🇬🇧Penarth, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom