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
Vertex Pharmaceuticals Incorporated
39 participants
Feb 27, 2023
INTERVENTIONAL
Conditions
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
Inclusion Criteria6
- 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%
Exclusion Criteria4
- 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
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Interventions
Oral inhalation using nebulizer.
Tablet for oral administration.
Locations(43)
View Full Details on ClinicalTrials.gov
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NCT05668741