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Active Not Recruiting
NCT05668741
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Conditions: Cystic Fibrosis
Sex: All
Ages: 18 Years – 65 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 26
Sponsor: Vertex Pharmaceuticals Incorporated
Location: University of Alabama at Birmingham - Child Health Research Unit Birmingham Alabama
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 Criteria
Key Inclusion Criteria:
* Body mass index is less 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 (\
Source: ClinicalTrials.gov (NCT05668741). StuddyBuddy aggregates publicly available trial information.