• VX09-809-102: A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects with Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation [Cohort 4]
Lumacaftor/ivacaftor (Kalydeco®) or placebo in Cystic Fibrosis patients 18 years and older heterozygous for the ?F508del mutation

The purpose of this research study is to see if the combination of two experimental drugs called lumacaftor and ivacaftor is safe to take and can help people with cystic fibrosis and one copy of the ?F508del mutation. This study uses a placebo, which looks like the lumacaftor/ivacaftor combination pill, but does not contain any medicine.

The study consists of 8 study visits at Massachusetts General Hospital over a 13 week period. Study procedures include:
• Lab work (urine and blood tests)
• PFTs (pulmonary function tests or spirometry)
• ECG (electrocardiogram)
• Sweat chloride tests
• Eye exams

Participants are assigned by chance to take twice a day for 56 days:
• Lumacaftor/ivacaftor or placebo

We are looking for cystic fibrosis patients who:
• Are 18 years old or older
• Have one copy of the ?F508del-CFTR mutation
• Are not pregnant or planning to become pregnant in the next 6 months, or planning to father a child in the next 6 months
• Are willing to attend all study visits and perform all study procedures and tests

Participants will be paid $1100 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, 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