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Home › Medications › ivacaftor

ivacaftor

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials of ivacaftor

31 trials tracked of ivacaftor: 20 in phase 3 or 4 and 10 with published results. The most-cited published study has 777 citations.

31Trials tracked
20Phase 3 & 4
0Recruiting
10With published results
Phase distribution
Phase 3 20 Phase 2 8 Phase 1 1 Other / NA 2
  1. Phase 3 A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor Completed · 777 cited
  2. Phase 3 A Phase 3 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-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation Completed · 511 cited
  3. Phase 3 A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis Completed · 285 cited
  4. Phase 3 Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation Completed · 224 cited
  5. Phase 3 Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation Completed · 165 cited
  6. Phase 3 Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation Completed · 138 cited
Show 25 more trials
  1. Phase 3 Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease Completed · 69 cited
  2. Phase 3 A 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 Completed · 35 cited
  3. Phase 3 A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation Completed · 32 cited
  4. Phase 3 A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor Completed · 18 cited
  5. Phase 3 An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations Completed
  6. Phase 3 A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF) Completed
  7. Phase 3 Rollover Study of VX-770 in Cystic Fibrosis Subjects Completed
  8. Phase 3 A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation Completed
  9. Phase 3 Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation Completed
  10. Phase 3 Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation Completed
  11. Phase 3 Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT) Completed
  12. Phase 3 A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation Completed
  13. Phase 3 Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation Completed
  14. Phase 3 Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation Completed
  15. Phase 2 Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia Completed
  16. Phase 2 Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation Completed
  17. Phase 2 Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function Completed
  18. Phase 2 A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation Completed
  19. Phase 2 Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation Completed
  20. Phase 2 A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation Completed
  21. Phase 2 Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion Completed
  22. Phase 2 Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation Completed
  23. Phase 1 TOPIC Trial for COPD Completed
  24. N/A Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients Completed
  25. N/A (Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil? Completed

Showing the 31 most-cited and recently-updated of 31 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

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Patient Mode — summaries use plain language, avoiding clinical jargon.
Review of CFTR modulator impact on cystic fibrosis outcomes from registry data
Pulmonology & Critical Care
Review of CFTR modulator impact on cystic fibrosis outcomes from registry data Cystic Fibrosis Patients See Lung Function Jump Over a Decade
This review and synthesis of Cystic Fibrosis Foundation Patient Registry data describes trends in adults with cystic fibrosis from 2011 to 2…
A decade of registry data shows lung function and weight improved for adults with cystic fibrosis as newer modulator therapies became availa…
medRxiv Apr 29, 2026