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Home › Conditions › Cystic fibrosis

Cystic fibrosis

7 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Cystic fibrosis

258 trials tracked for Cystic fibrosis: 113 in phase 3 or 4 and 53 with published results. The most-cited published study has 2131 citations.

258Trials tracked
113Phase 3 & 4
0Recruiting
53With published results
Phase distribution
Phase 4 26 Phase 3 87 Phase 2 76 Phase 1 13 Other / NA 56
  1. Phase 3 A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Completed · 2,131 cited
  2. Phase 3 A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F) Completed · 1,381 cited
  3. 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 · 786 cited
  4. 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 · 516 cited
  5. Phase 3 Study of Ataluren (PTC124™) in Cystic Fibrosis Completed · 337 cited
  6. Phase 3 A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation Completed · 296 cited
Show 44 more trials
  1. Phase 3 Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age Completed · 294 cited
  2. Phase 3 A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Completed · 277 cited
  3. Phase 3 A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation Completed · 272 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 · 167 cited
  6. Phase 3 A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del Completed · 143 cited
  7. Phase 3 Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation Completed · 140 cited
  8. Phase 3 Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del Completed · 134 cited
  9. Phase 3 Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years Completed · 129 cited
  10. Phase 3 Long Term Administration of Inhaled Mannitol in Cystic Fibrosis Completed · 121 cited
  11. Phase 3 OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis Completed · 98 cited
  12. Phase 3 A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) Completed · 97 cited
  13. Phase 3 A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation Completed · 94 cited
  14. Phase 4 Treatment of Aspergillus Fumigatus (a Fungal Infection) in Patients With Cystic Fibrosis Completed · 85 cited
  15. Phase 3 A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy Completed · 71 cited
  16. Phase 3 A Study Evaluating the Long-term Safety of VX-445 Combination Therapy Completed · 71 cited
  17. Phase 3 A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation Completed · 71 cited
  18. Phase 3 Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation Completed · 71 cited
  19. Phase 3 Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older Completed · 71 cited
  20. Phase 3 A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes Completed · 71 cited
  21. Phase 3 Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy Completed · 71 cited
  22. Phase 3 A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis Completed · 71 cited
  23. Phase 3 Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del Completed · 71 cited
  24. Phase 3 A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy Completed · 71 cited
  25. Phase 3 Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease Completed · 69 cited
  26. Phase 3 Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) Completed · 66 cited
  27. Phase 3 Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF Completed · 58 cited
  28. Phase 3 A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF) Completed · 55 cited
  29. Phase 3 A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation Completed · 52 cited
  30. Phase 4 Population Pharmacokinetics and Safety of Intravenous Ceftolozane/Tazobactam in Adult Cystic Fibrosis Patients Completed · 49 cited
  31. Phase 4 Steady-state Pharmacokinetics of Ceftazidime/Avibactam in Cystic Fibrosis Completed · 48 cited
  32. Phase 3 Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients Completed · 47 cited
  33. Phase 3 SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis Completed · 43 cited
  34. Phase 3 A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation Completed · 42 cited
  35. Phase 3 Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor Completed · 42 cited
  36. 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
  37. 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 · 33 cited
  38. Phase 3 Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) Infection Completed · 26 cited
  39. Phase 4 Comparison of Airway Clearance Therapy in Cystic Fibrosis Using the Same VEST Therapy Device But With Different Settings Completed · 25 cited
  40. Phase 4 A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation Completed · 23 cited
  41. Phase 4 Ease of Use and Microbial Contamination of Tobramycin Inhalation Powder (TIP) Versus Nebulised Tobramycin Inhalation Solution (TIS) and Nebulised Colistimethate (COLI) Completed · 19 cited
  42. Phase 3 Open Label Extension to Bridging Study CTBM100C2303 Completed · 19 cited
  43. Phase 3 A Safety and Efficacy Trial of Inhaled Mannitol in Adult Cystic Fibrosis Subjects Completed · 18 cited
  44. 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

Showing the 50 most-cited and recently-updated of 258 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.

What the trials found Updated — new results For clinicians

Cystic fibrosis: what the trials found

New evidence includes the use of Omadacycline Injection (Nuzyra) 1, Semaglutide 3, and Mannitol Inhalant Product, which showed a statistically significant improvement in Forced Expiratory Volume in One Second (FEV1) Percent of Predicted (46.9, p=0.04), though it did not significantly impact scores on the CFQR-R (p=0.94) or CFRSD-CRISS (p=0.13) scales 2.

Established treatments for cystic fibrosis include various antimicrobial and supportive therapies such as Itraconazole 7, Ceftolozane/Tazobactam, which demonstrated a 97.1% probability of target attainment at 8 mcg/ml 8, and Ceftazidime/avibactam 9. Other established interventions include Tobramycin Inhalation Powder 12, Colistin 16, Tedizolid PO 21, Liprotamase 22, Vitamin D3 14, and Pancrelipase 15.

Management of gastrointestinal symptoms has been evaluated with CREON, which showed consistent mean changes in Total Symptom Score (TSS), Abdominal Symptom Domain Score (ASDS), and Bowel Movement Symptom Score (BMSS) across multiple follow-up intervals 13. High-pressure/variable-frequency HFCWC settings were shown to significantly reduce both wet and dry sputum weight 10.

Clinical trials for azithromycin showed no significant changes in lung function (p=0.51), CFRSD-CRISS scores (p=0.17), or CFQ-R RSS scores (p=0.56) 17. Additionally, LUM/IVA did not result in statistically significant changes in maximal oxygen consumption (p=0.3021) or exercise duration during cardiopulmonary exercise testing at week 24 11.

Recent results — preliminary, needs further review

  • Brensocatib (Phase 2) 4
  • MRT5005 (Phase 2), which showed various changes in ppFEV1 from baseline ranging from -2.7017 to 5.1117 5
  • Volara System (Phase N/A) 6

For the clinician treating this condition

  • Mannitol Inhalant Product significantly improves FEV1% predicted, though it does not demonstrate statistically significant improvements in patient-reported respiratory symptom scores.
  • HFCWC with higher pressure/variable-frequency settings are effective for reducing sputum weight.
  • Ceftolozane/Tazobactam shows a high probability of target attainment (97.1%) at 8 mcg/ml.
  • Azithromycin and LUM/IVA did not demonstrate statistically significant improvements in primary lung function or exercise capacity metrics in the studied cohorts.

AI synthesis of 19 cited trials, updated Jun 23, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Obstructive Sleep Apnea Affects Nearly Half of Cystic Fibrosis Patients
Pediatrics Meta-analysis
Obstructive Sleep Apnea Affects Nearly Half of Cystic Fibrosis Patients Nearly half of people with cystic fibrosis have sleep apnea
A meta-analysis finds that 48% of cystic fibrosis patients have obstructive sleep apnea, linked to worse lung function and oxygen levels.
Almost half of all people with cystic fibrosis also live with obstructive sleep apnea, a condition that can impact oxygen and lung function.
NEJM Jul 18, 2026
Ultrasound Reveals Reduced Quadriceps Muscle in Cystic Fibrosis
Radiology & Imaging Meta-analysis
Ultrasound Reveals Reduced Quadriceps Muscle in Cystic Fibrosis Ultrasound Shows Muscle Loss in People with Cystic Fibrosis
Meta-analysis shows significantly reduced rectus femoris thickness in people with cystic fibrosis, with moderate correlations to lung functi…
New analysis shows that people with cystic fibrosis have significantly thinner thigh muscles compared to healthy individuals.
Jun 17, 2026
Quadruple antimycobacterial regimen achieves clinical stability in cystic fibrosis patient with M. abscessus
Pediatrics Sys. Review
Quadruple antimycobacterial regimen achieves clinical stability in cystic fibrosis patient with M. abscessus Case report shows quadruple regimen for cystic fibrosis infection
A case report and literature review describes a 21-year-old man with cystic fibrosis and Mycobacterium abscessus infection who achieved clin…
A young man with cystic fibrosis and a hard-to-treat infection achieved stability using a four-drug antimycobacterial regimen.
Frontiers Jun 2, 2026
Narrative review finds insufficient evidence for CFTR modulator monotherapy in cystic fibrosis with F508del homozygosity
Pediatrics Sys. Review
Narrative review finds insufficient evidence for CFTR modulator monotherapy in cystic fibrosis with F508del homozygosity New review shows single drug therapy does not help people with cystic fibrosis who have two F508del gene variants
This narrative review synthesized evidence from 10 early-phase trials on CFTR modulator monotherapy for cystic fibrosis with F508del homozyg…
A review of early studies found that taking one CFTR modulator drug alone does not improve survival, lung function, or quality of life for p…
Cochrane May 13, 2026
Expert consensus guidelines for N-acetylcysteine use in various respiratory conditions
Rheumatology Guideline
Expert consensus guidelines for N-acetylcysteine use in various respiratory conditions Experts share new guidance on using N-acetylcysteine for lung health
This expert consensus guideline reviews the clinical utility of N-acetylcysteine (NAC) across several respiratory diseases. The authors sugg…
Medical experts have reached a consensus on how a specific supplement might help people managing various chronic lung diseases.
Frontiers May 4, 2026
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
Systematic review of lung transplantation in cystic fibrosis with nontuberculous mycobacteria infection
Nutrition & Obesity Medicine Sys. Review
Systematic review of lung transplantation in cystic fibrosis with nontuberculous mycobacteria infection Lung Transplant for Cystic Fibrosis Patients with NTM Infection: What New Data Reveals
This systematic review synthesizes evidence from four retrospective studies on lung transplantation in people with cystic fibrosis and nontu…
For people with cystic fibrosis who need a lung transplant, having a specific lung infection called NTM makes decisions harder, but new data…
Cochrane Apr 23, 2026

Questions about Cystic fibrosis

How have cystic fibrosis patient outcomes changed in adults from 2011 to 2022?

From 2011 to 2022, adults with cystic fibrosis saw major improvements in lung function, BMI, and fewer exacerbations, largely due to CFTR modulator therapies like elexacaftor/tezacaftor/ivacaftor.

Full answer →