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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
26
Oral inhalation using nebulizer.
Tablet for oral administration.
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 Through Week 8 [SAD and MAD]
MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 Through Safety Follow-up Visit [up to Week 28]
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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
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
...and 16 more locations