An Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

VX16-809-121

This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).

2018-08-10

2020-10-09

2021-10-07

Completed

Early phase I

51

Key Inclusion Criteria: * Subjects with confirmed diagnosis of CF. * Homozygous for F508del (F/F). * Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit. Key Exclusion Criteria: * Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject. * Solid organ or hematological transplantation. * History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study. Other protocol defined Inclusion/Exclusion criteria may apply.

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2022-11-02