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Cystic Fibrosis: early-phase trial registry
49 of 170 indexed Cystic Fibrosis trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #64 of 28,707 conditions by registered trial count.
170 US clinical trials · 49 currently recruiting · #64 of 28,707 conditions by registered trial count
Cystic Fibrosis: 170 US clinical trials tracked, 49 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.
Enrollment posture brief
Registry condition cystic-fibrosis
OPEN 49 | RECR 29% | LATE 34 | EARLY 61 | RANK #64 | SPON 20
Volume-matched condition peers
Nearest open-share peer: Prostatic Neoplasms (29% recruiting · 66 trials)
Cystic Fibrosis lists 49 open slots (29% of 170 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Cystic Fibrosis on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Cystic Fibrosis is recruiting below its size-band peers.
49 of 170 indexed trials (28.8%) are open to enrollment, against a 37.1% average across 139 conditions with 100+ trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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Sponsorship of Cystic Fibrosis trials is spread across many organizations.
Vertex Pharmaceuticals Incorporated accounts for 20 of 170 trials (11.8%). 84 distinct sponsors appear on at least one study for this condition label.
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Most Cystic Fibrosis trials plan to enrol fewer than 100 people.
Median target enrolment is 59 among the 170 trials that report a genuine target (sentinel values excluded). 107 of those (62.9%) plan for fewer than 100 participants.
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Cystic Fibrosis research is mostly interventional.
121 interventional and 49 observational studies make up this index (71.2% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and 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
Named interventions
Most-linked intervention names on Cystic Fibrosis records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Cystic Fibrosis trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Cystic Fibrosis page
170 registered trials, 49 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 49 Cystic Fibrosis trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Vertex Pharmaceuticals Incorporated sponsors the most Cystic Fibrosis trials on record, review their full trial history before evaluating a specific study. See Vertex Pharmaceuticals Incorporated's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Cystic Fibrosis condition card totals 170 registered trials; most-linked intervention Placebo; top US site state Colorado. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.