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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

A Phase 1/2 Dose Escalation Study Evaluating the Safety, and Tolerability and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - 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 Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

VX-522 mRNA therapy

Oral inhalation using nebulizer.

DRUG

IVA

Tablet for oral administration.

Locations (20)

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