Cystic Fibrosis Research & Innovation

The University of Michigan Cystic Fibrosis Center is advancing the field of CF treatment through a wide range of clinical studies and basic research endeavors.

Cystic Fibrosis Clinical Studies 

Current multicenter Cystic Fibrosis Foundation-endorsed studies underway at U-M include:

  1. A 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
  2. VX14-661-108 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.
  3. VX14-661-107 A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR).
  4. VX14-661-109: A Phase 3, Randomized, Double-Blind, Ivacaftor Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of 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 CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive.
  5. VX14-661-110 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 (VX14-661-110).
  6. An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis.
  7. A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients with Cystic Fibrosis
  8. GOAL-e² (G551D Observational Study – Expanded to Additional Genotypes and Extended for Long Term Follow-Up).
  9. Optimizing Treatment for Early Pseudomonas aeruginosa Infection in Cystic Fibrosis.
  10. Qualitative Research in Cystic Fibrosis for the Development of a New Patient Reported Outcome (PRO) Measure.
  11. The EPIC Observational Study: Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children with CF (Years 11-15).
  12. A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across sponsors.
  13. A Multi-Center Study of A New Method of Sweat Testing: The CF Quantum® Sweat Test (CFQuantum003).

Cystic Fibrosis Basic Research 

Several laboratories at the University of Michigan are involved in cutting edge research on CF treatment and basic biology.  These include:

  • Research led by Dr. John Lipuma M.D. exploring molecular epidemiology of respiratory tract infections in CF.   The Lipuma Lab serves as a national reference laboratory for the Cystic Fibrosis Foundation, and maintains an extensive culture collection that includes approximately 40,000 bacterial strains.
  • Research led by Dr. Lindsey Caverly M.D. investigating the molecular epidemiology of atypical mycobacterial infection in CF.