National Jewish Health | Clinical Research Services, Denver, CO
Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
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.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
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: ≥ 40% to less than or equal to (≤) 90%
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
33 participants in 3 patient groups
Loading...
Central trial contact
Medical Information
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal