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This study examined a combination therapy containing VX-121 in patients with cystic fibrosis. It enrolled patients who were homozygous for F508del in the CFTR gene, heterozygous for F508del with a gating or residual function mutation, or who carried certain CFTR mutations known to respond to treatment. It was a Phase 3 study and is completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
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Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * Participant has one of the following genotypes: * Homozygous for F508del; * Heterozygous for F508del and a gating (F/G) mutation; * Heterozygous for F508del and a residual function (F/RF) mutation; * At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation * Forced expiratory volume in 1 second (FEV1) value \>=40% and \<=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 \>=40% and \<=80% for participants not currently receiving CFTR protein modulator therapy Key Exclusion Criteria: * 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) * Lung infection with organisms associated with a more rapid decline in pulmonary status * Pregnant or breast-feeding females Other protocol defined Inclusion/Exclusion criteria may apply.