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
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