Cystic Fibrosis / Bronchiectasis

  • A Prospective, 5-Year Registry Study to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa (PA) Isolates from Patients with CF in the US (AIR-CF5).
    PI: Emily DiMango, MD
    Contact: Surinder Narula (212) 305-0251
  • A phase 2, randomized, double blind, placebo-controlled study of AeroVanc for the treatment of persistent methicillin-resistant Staphylococcus aureus lung infection in cystic fibrosis patients. Vancomycin is an effective antibiotic for the treatment of MRSA. Presently, this antibiotic is only available intravenously for the treatment of lung infections. When given as an IV infusion, Vancomycin can be associated with kidney damage and hearing problems. To reduce the risk of the side effects, there is potentially a significant advantage in delivering vancomycin directly to the the lung. AeroVanc is a novel dry powder formulation of vancomycin in intended for the treatment of MRSA lung infection in CF patients. This formulation is intended for self-administration at home, is easy to use, and requires much less drug than an iv administration and only a fraction of the time to administer. Potential side effects include allergic reaction, low oxygen levels and asthma attacks. This study is the first randomized placebo-controlled Phase 2a study to evaluate the effectiveness and safety of AeroVanc in CF patients with persistent MRSA lung infection.
    PI: Emily DiMango, MD
    Contact: Surinder Narula (212) 305-0251
  • Title: A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX 661 in Combination with Ivacaftor for 12Weeks in Subjects with Cystic Fibrosis, Homozygous for the F508del CFTR Mutation Description: Cystic Fibrosis (CF) is the most common fatal genetic disease in persons of European descent. CF is an autosomal recessive genetic disease caused by a defect in the gene encoding the CF transmembrane conductance regulator (CFTR) – an epithelial chloride ion (Cl-) channel activated by cyclic AMP-dependent protein kinase A that is responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. This function is defective in CF patients due to a loss of either cell surface expression and/or function. The most prevalent CFTR mutation is the F508del-CFTR mutation. The F508del-CFTR mutation interferes with the ability of the CFTR protein to reach and remain at the cell surface, as well as to open and close, resulting in decreased Cl- transport. Two complementary approaches to increase CFTR-mediated Cl- secretion in airway epithelia have been studied. One approach is to treat with a compound that will modify the cellular processing and delivery of CFTR protein to the cell surface. This kind of compound has been termed a CFTR corrector. A second approach is to treat with a compound that increases channel gating activity of protein kinase A-activated CFTR at the cell surface to enhance ion transport. This kind of compound has been termed a potentiator. VX-661 is a compound developed by Vertex that has been shown to have CFTR corrector properties, while Ivacaftor (VX-770) has been shown to have CFTR potentiator properties. This multi-center, 12-week study is designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of VX-661 in combination with Ivacaftor.
    PI: Claire Keating, MD
    Contact: Kristina Rivera (212) 305-4675
  • Title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment with Lumacaftor in Combination with Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Description: Cystic Fibrosis (CF) is the most common fatal genetic disease in persons of European descent. CF is an autosomal recessive genetic disease caused by a defect in the gene encoding the CF transmembrane conductance regulator (CFTR) – an epithelial chloride ion (Cl-) channel activated by cyclic AMP-dependent protein kinase A that is responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. This function is defective in patients with CF due to a loss of either cell surface expression and/or function. The most prevalent CFTR mutation is the F508del-CFTR mutation. The F508del-CFTR mutation interferes with the ability of the CFTR protein to reach and remain at the cell surface, as well as to open and close, resulting in decreased Cl- transport. Two complementary approaches to increase CFTR-mediated Cl- secretion in airway epithelia have been studied. One approach is to treat with a compound that will modify the cellular processing and delivery of CFTR protein to the cell surface. This kind of compound has been termed a CFTR corrector. A second approach is to treat with a compound that increases channel gating activity of protein kinase A-activated CFTR at the cell surface to enhance ion transport. This kind of compound is termed a potentiator. Lumacaftor (VX-809) is a compound developed by Vertex that has shown to have CFTR corrector properties, while Ivacaftor (VX-770) has shown to have CFTR potentiator properties. Study VX12-809-104 and study VX09-809-102 were designed to evaluate the efficacy of Lumacaftor in combination with Ivacaftor. This trial is the rollover protocol for the two studies. This is a Phase 3, parallel-group, multicenter, rollover study for subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in Study 103, Study 104, or Cohort 4 of Study 102. Study 105 is designed to evaluate the safety and efficacy of long-term treatment of Lumacaftor in combination with Ivacaftor.
    PI: Emily DiMango, MD
    Contact: Eva Aquino (212) 305-4675