A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
Study ID: STU-2019-1580
Summary
Primary endpoint Safety and tolerability assessments based on adverse events (aes), clinical laboratory (serum chemistry and hematology), standard 12-lead eCGs, vital signs, pulse oximetry, and ophthalmological examinations (oes) Secondary endpoint absolute change from baseline in sweat chloride exploratory endpoints * absolute change from baseline in the following growth parameters: o Body mass index (BMi)-for-age z-score and BMi o Weight-for-age z-score and weight o Length/height-for-age z-score and length/height o Weight-for-length z-score * absolute change from baseline in the following markers of pancreatic function: o Fecal elastase-1 (Fe-1) levels o Serum immunoreactive trypsin and trypsinogen (iRT) levels * absolute change from baseline in fecal calprotectin, a marker of intestinal inflammation
Primary objective To evaluate the safety and tolerability of long-term lumacaftor/ivacaftor (LuM/iVa) treatment in subjects with cystic fibrosis (CF), who are homozygous for F508del and 12 to [Less Than]24 months of age at treatment initiation Secondary objective To evaluate the pharmacodynamics (PD) of long-term LuM/iVa treatment in subjects with CF, who are homozygous for F508del and 12 to [Less Than]24 months of age at treatment initiation