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Cystic Fibrosis clinical trials
49 of 170 indexed Cystic Fibrosis trials are currently recruiting.
170 US clinical trials · 49 currently recruiting
Cystic Fibrosis is the subject of 170 registered US clinical trials on ClinicalTrials.gov, 49 of them currently open to new participants. 34 are in Phase 3-4 (later-stage) and 61 in Phase 1-2 (earlier-stage). The most active sponsor is Vertex Pharmaceuticals Incorporated, running 20 of these trials.
The research picture
Cystic Fibrosis has 170 registered US clinical trials, 49 of them open to new participants right now, about 29% of the total.
- 49
- recruiting participants now
- 29%
- of trials open to enrollment
- 34
- in Phase 3–4 (later-stage)
- 20
- top sponsor: Vertex Pharmaceuticals Incorporated
Active & Recent Trials
170 total, page 2 of 4
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
Vertex Pharmaceuticals Incorporated
NCT05444257
Gene Modifiers of Cystic Fibrosis Lung Disease
University of North Carolina, Chapel Hill
NCT00037765
Colorectal Cancer Screening in Cystic Fibrosis
University of Washington, the Collaborative Health Studies Coordinating Center
NCT05362344
Strength and Muscle Related Outcomes for Nutrition and Lung Function in CF
Jaeb Center for Health Research
NCT05639556
Prospective Study of Pregnancy in Women With Cystic Fibrosis
Amalia Magaret
NCT04828382
The PROMISE Pediatric Study 6 to 11 Years Old
Nicole Hamblett
NCT04613128
Genetic Modifiers of Cystic Fibrosis (CF) Liver Disease
University of North Carolina, Chapel Hill
NCT00804583
Lung Transplant READY CF 2: A Multi-site RCT
University of Washington
NCT06030206
Host and Bacterial Mechanisms During Cystic Fibrosis Pulmonary Exacerbations
National Jewish Health
NCT04354038
Pulmonary Vascular Disease in CF
Children's Hospital Medical Center, Cincinnati
NCT04549077
Hyperpolarized Imaging for New Treatments
Children's Hospital Medical Center, Cincinnati
NCT04259970
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Indiana University
NCT04206436
High Resolution Micro OCT Imaging
University of Alabama at Birmingham
NCT03256773
Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI
Children's Hospital Medical Center, Cincinnati
NCT02848560
Non-contrast Lung Perfusion Mapping Applied for New Insights in Cystic Fibrosis
Children's Hospital Medical Center, Cincinnati
NCT04467957
Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants
Vertex Pharmaceuticals Incorporated
NCT07349394
Effect of Losartan in Cystic Fibrosis (CF)-NIH Grant #133240
University of Kansas Medical Center
NCT03435939
Mechanisms for Vascular Dysfunction and Exercise Tolerance in CF
Augusta University
NCT02690064
Implementation of a Clinical Tool to Improve Waitlist Mortality in Patients With Cystic Fibrosis
The Cleveland Clinic
NCT04687475
SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes
University of Minnesota
NCT06149793
HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy
Indiana University
NCT04798014
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
Novartis Pharmaceuticals
NCT00388505
Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis
Boehringer Ingelheim
NCT00737100
A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
University of Alabama at Birmingham
NCT02477319
MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis
Amgen
NCT01180634
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
Syntara
NCT00630812
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Vertex Pharmaceuticals Incorporated
NCT01225211
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
PTC Therapeutics
NCT02139306
Early Intervention in Cystic Fibrosis Exacerbation
Johns Hopkins University
NCT01104402
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
Vertex Pharmaceuticals Incorporated
NCT02392234
Study of Ataluren (PTC124™) in Cystic Fibrosis
PTC Therapeutics
NCT00803205
Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions
Duke University
NCT03938324
OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis
Bonnie Ramsey
NCT02054156
Open-Label Phase III Long-Term Safety Trial of Liprotamase
Anthera Pharmaceuticals
NCT00449904
Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis
Proteostasis Therapeutics
NCT03140527
Liprotamase Efficacy Trial in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
Anthera Pharmaceuticals
NCT00449878
Saline Hypertonic in Preschoolers
University of Washington, the Collaborative Health Studies Coordinating Center
NCT02378467
Utility of CD64 and TLR2 Assays to Diagnose Acute Pulmonary Exacerbations in Cystic Fibrosis
National Jewish Health
NCT04397809
Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation
Nivalis Therapeutics
NCT02589236
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
Vertex Pharmaceuticals Incorporated
NCT01707290
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
NCT03224351
Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation
University of Colorado, Denver
NCT00788359
G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)
Steven M Rowe
NCT01521338
T Cell Memory Fuels the Innate Response in Chronic CF Lung Disease
National Jewish Health
NCT04397861
Study of LYM-X-SORB™ to Improve Fatty Acid and Choline Status in Children With CF and PI
Avanti Polar Lipids
NCT00406536
Diabetes Therapy to Improve BMI and Lung Function in CF
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NCT00072904
Feasibility of a Mobile Medication Plan Application in CF Patient Care
Boston Children's Hospital
NCT03637504
Aztreonam for Inhalation (AI) in Patients With Cystic Fibrosis & P. Aeruginosa Infection
Gilead Sciences
NCT01055847
Cystic Fibrosis - Insulin Deficiency, Early Action
Sydney Children's Hospitals Network
NCT01100892
An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
NCT01863238
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 34 |
| Phase 2 | 27 |
| Phase 3 | 19 |
| Phase 4 | 15 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Cystic Fibrosis Trial Landscape
170 indexed Cystic Fibrosis studies · 49 recruiting (29%).
Phase mix: 34 late (III/IV) vs 61 early (I/II).
Top sponsor: Vertex Pharmaceuticals Incorporated (20 trials), 9 others listed. 50 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Cystic Fibrosis?
PlainTrial tracks 170 US clinical trials for Cystic Fibrosis, of which 49 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Cystic Fibrosis?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.