Protocol Information

If you are interested in learning more about this study, please call or email the contact person below

Title VX15-809-115: A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Description Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

Study visits will be in downtown Buffalo
Objective This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of Lumacaftor/Ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with Cystic Fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if patient meet the eligibility criteria.

Principal Investigator GOETZ, DANIELLE
Age Group Children
Drugs Involved
Clinical Trials.gov http://www.clinicaltrials.gov/ct2/show/NCT02797132/
Status Open
Key Eligibility Children Aged 2- 5 Years
Cystic Fibrosis
Homozygous for the F508del-CFTR Mutation
Disease Group Children's Health/Pediatrics; Cystic Fibrosis
Treatment Lumacaftor/Ivacaftor
Contact BETH CAHILL
716-878-7561
bcahill@upa.chob.edu