Active Research Studies

Current Research on Cystic Fibrosis

Listed below are many of our active cystic fibrosis studies:

  • Normalized T1 MRI to Detect and Monitor Early-Stage Cystic Fibrosis Lung Disease Progression
  • OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis: The OPTIMIZE Multicenter, Placebo-Controlled, Double-Blind, Randomized Trial
  • PTC Therapeutics: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Cystic Fibrosis
  • PTC Therapeutics: A Phase 3 Extension Study of Ataluren (PTC124) In Patients with Nonsense Mutation Cystic Fibrosis
  • PTC Therapeutics: An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124)
  • A Two-Part Multicenter Prospective Longitudinal Study of CFTR-Dependent Disease Profiling In Cystic Fibrosis (PROSPECT)
  • Saline Hypertonic In Preschoolers (SHIP)
  • Vertex Pharmaceuticals Inc: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
  • Vertex Pharmaceuticals: A Phase 3, Open-Label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
  • Vertex Pharmaceuticals Inc.: A Phase 3, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
  • Vertex Pharmaceuticals: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
  • Anthera Pharmaceuticals Inc: Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency [AN-EPI3331]
  • Corbus Pharmaceuticals Inc.: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis
  • Alcresta Pharmaceuticals: Study to Evaluate Safety, Tolerability and Fat Absorption of a Novel Enteral Feeding In-line Digestive Enzyme Cartridge (EFIC™) in Cystic Fibrosis Patients Using Enteral Tube Feeding For 24-Hour Plasma Pharmacodynamic Fatty Acid Profile
  • Nivalis Therapeutics, Inc.: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients with Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation and Being Treated with Lumacaftor/Ivacaftor
  • A Phase I, Single Center, Open Label, Single Dose, Dose Escalation Study Assessing the Safety and Tolerability of Allogeneic Mesenchymal Stem Cell Transplantation in Adults with Cystic Fibrosis

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