A Phase 3, 2-Arm, Open-Label Study to Evaluate the Safety & Pharmacodynamics of Long-Term Ivacaftor

Clinical Trial

Offered by: Nemours Children's
Location: Delaware Valley

Trial Name

A Phase 3, 2-Arm, Open-Label Study to Evaluate the Safety & Pharmacodynamics of Long-Term Ivacaftor

What is the trial about?

The purpose of this study is to learn more about the safety and effects of long-term ivacaftor treatment on children with certain forms of cystic fibrosis (CF) who are less than 24 months of age when treatment begins. We will also look at how long-term ivacaftor may affect your child’s body.

Who can participate?

Children under two years of age that have CF, caused by one of a specific group of genetic mutations.

What is involved?

The study will take 120 weeks (about 2.5 years). This includes a 2-year treatment period and a follow-up eye exam 6 months after the last dose of the study drug.

There will be up to 16 clinic visits, one eye doctor appointment and one telephone call. Your child will also have up to seven eye examinations during scheduled visits.

Blood draws: The total amount of blood that will be taken from your child during this study will be about 69 mL (or about 14 teaspoons).  Total of 14 scheduled blood draws.

Electroencephalograms (ECG): Total of 13 scheduled ECGs.