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
Vertex Pharmaceuticals Incorporated
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.
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugVX-522 mRNA therapy
Oral inhalation using nebulizer.
- DrugIVA
Tablet for oral administration.
Locations (43)
- University of Alabama at Birmingham - Child Health Research UnitBirmingham, Alabama
- Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long BeachLong Beach, California
- Stanford University - Palo Alto - PulmonologyPalo Alto, California
- National Jewish HealthDenver, Colorado
- Shands Hospital - PulmonologyGainesville, Florida
- Northwestern Memorial Hospital - Feinberg PavilionChicago, Illinois