• Vertex 108: A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects with Cystic Fibrosis Who are 2 Through 5 Years of Age and Have a CFTR Gating Mutation
Ivacaftor (Kalydeco®) in Cystic Fibrosis patients 2 to 5 years old with the G551D mutation

The purpose of this research study is to see if ivacaftor can help children 2-5 years of age with cystic fibrosis and the G551D mutation.

The study consists of 6 study visits at Massachusetts General Hospital over a 16 week period. Study procedures include:
• Blood and urine tests
• Electrocardiograms (ECGs)
• Eye exams
• Follow up telephone calls from study staff

During the study, we will give you ivacaftor that you will give to your child, with the help of the study doctor.

Your child will be paid $650 for completing the study. Out of pocket expenses can be reimbursed. The study drug is being provided at no cost to you.

For more information, please contact Caitlin Doolittle at cdoolittle1@partners.org or 617-726-3719.
Institution  –  MGH - Massachusetts General Hospital, Boston, MA
Principal Investigator  – Allen Lapey, MD
  
Enrollment Information
For further information about enrolling a patient in this trial, contact the person below.
Name  – Caitlin Doolittle
Phone  – 617-726-3719