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Cystic Fibrosis clinical trials
Every US clinical trial registered for Cystic Fibrosis — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
170 US clinical trials · 49 currently recruiting
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
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
Role of Genetic Factors in the Development of Lung Disease
National Heart, Lung, and Blood Institute (NHLBI)
NCT00001532
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
xCures
NCT06539169
Natural History of Bronchiectasis
National Heart, Lung, and Blood Institute (NHLBI)
NCT00943514
Acoustic Waveform Respiratory Evaluation
Indiana University
NCT06512064
Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study
Chris Goss
NCT05548283
Genetics of Insulin and Incretins in Cystic Fibrosis
Children's Hospital of Philadelphia
NCT01852448
Rare CFTR Mutation Cell Collection Protocol (RARE)
George Solomon
NCT03161808
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
University of Washington, the Collaborative Health Studies Coordinating Center
NCT06654752
A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Nicole Hamblett
NCT06504589
Natural History of Noncirrhotic Portal Hypertension
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NCT02417740
Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease
Mario Castro, MD, MPH
NCT04624490
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
Vertex Pharmaceuticals Incorporated
NCT06154447
Exercise in Child Health
University of California, Irvine
NCT05359991
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Vertex Pharmaceuticals Incorporated
NCT05422222
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Sonya Heltshe
NCT04509050
Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
Boston Children's Hospital
NCT06683092
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
ReCode Therapeutics
NCT06237335
Impact of Sinus Surgery on Individuals With Cystic Fibrosis
University of California, Los Angeles
NCT04469439
Remote Sputum Collection in Cystic Fibrosis
University of Pennsylvania
NCT06950892
Health Outcomes of Parents With Cystic Fibrosis-Aim 2
University of Pittsburgh
NCT06296394
Lung Transplant READY CF 2: CARING CF Ancillary RCT
University of Washington
NCT06032273
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants
Vertex Pharmaceuticals Incorporated
NCT07283770
Markers of Osteoporosis in Cystic Fibrosis
University of Texas Southwestern Medical Center
NCT03921060
Regional Phenotyping of CF and Non-CF Bronchiectasis
Children's Hospital Medical Center, Cincinnati
NCT04793867
Role of Body Fat Distribution in Metabolic and Pulmonary Decline in Cystic Fibrosis (ORBIT-CF)
Emory University
NCT04002882
Sinus Disease in Young Children With Cystic Fibrosis
University of California, Los Angeles
NCT06191640
Estrogen Supplementation and Bone Health in Women With CF
Johns Hopkins University
NCT05704036
Hormonal Responses to a Mixed Meal in People With Cystic Fibrosis
University of Cincinnati
NCT06163482
FEED-Cystic Fibrosis (FEED-CF)
Emory University
NCT05766774
Regional Monitoring of CF Lung Disease
Children's Hospital Medical Center, Cincinnati
NCT06339593
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
Children's Hospital Medical Center, Cincinnati
NCT04580368
Beta-cell Response to Incretin Hormones in Cystic Fibrosis
University of Pennsylvania
NCT01851694
BEGIN Novel ImagiNG Biomarkers
Children's Hospital Medical Center, Cincinnati
NCT05517655
Targeting Inflammation With Losartan to Improve Response to Modulator Therapy in Cystic Fibrosis.
University of Kansas Medical Center
NCT06364176
A Pilot and Feasibility Study to Evaluate High vs Low Glycemic Index Mixed Meal Tolerance Test in Adolescents and Young Adults With Cystic Fibrosis
Emory University
NCT06350149
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Vertex Pharmaceuticals Incorporated
NCT05668741
Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis
Massachusetts General Hospital
NCT06216704
Impact of Highly Effective Modulator Therapy on the Cystic Fibrosis Microbiome
Dartmouth-Hitchcock Medical Center
NCT05982795
Skin-interfaced Colorimetric Bifluidic Sweat Sensor Device for the Diagnosis of Cystic Fibrosis (CF)
Milton S. Hershey Medical Center
NCT05998629
Continuous Glucose Monitoring and OGTT Screen for Cystic Fibrosis Related Diabetes in Cystic Fibrosis
Medical College of Wisconsin
NCT06560463
Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis
University of North Carolina, Chapel Hill
NCT06057714
GLP-1 Agonist Therapy in Cystic Fibrosis-Related Glucose Intolerance
University of Pennsylvania
NCT04731272
Assessing Tenapanor as a Treatment of CF-related Constipation.
Massachusetts General Hospital
NCT06810167
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis
SpliSense
NCT06429176
Personalized Theratyping Trial
George Solomon
NCT03587961
A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)
Spirovant Sciences
NCT06526923
The Effects of a Low Glycemic Load Diet on Dysglycemia and Body Composition in Adults With Cystic Fibrosis-Related Diabetes
Rhode Island Hospital
NCT05723445
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
Krystal Biotech
NCT05504837
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
Arcturus Therapeutics
NCT06747858
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes
Nicole Hamblett
NCT03350828
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
ClinicalTrials.gov lists 170 US studies indexed under Cystic Fibrosis, and 49 of those are currently open to recruitment — roughly 29% of the total volume on the registry. 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.
The phase distribution for Cystic Fibrosis shows 34 late-stage studies (Phase 3 and Phase 4 combined) alongside 61 earlier-phase entries (Phase 1 through Phase 2). 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.
Top sponsor activity for Cystic Fibrosis is led by Vertex Pharmaceuticals Incorporated with 20 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 100 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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.