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ClinicalTrials.gov 49 recruiting now official registry

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

RECRUITING 3,500 participants

Role of Genetic Factors in the Development of Lung Disease

National Heart, Lung, and Blood Institute (NHLBI)

NCT00001532

RECRUITING 1,000 participants

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

xCures

NCT06539169

RECRUITING 900 participants

Natural History of Bronchiectasis

National Heart, Lung, and Blood Institute (NHLBI)

NCT00943514

RECRUITING NA 800 participants

Acoustic Waveform Respiratory Evaluation

Indiana University

NCT06512064

RECRUITING Phase 4 730 participants

Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study

Chris Goss

NCT05548283

RECRUITING 550 participants

Genetics of Insulin and Incretins in Cystic Fibrosis

Children's Hospital of Philadelphia

NCT01852448

RECRUITING 500 participants

Rare CFTR Mutation Cell Collection Protocol (RARE)

George Solomon

NCT03161808

RECRUITING NA 430 participants

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics

University of Washington, the Collaborative Health Studies Coordinating Center

NCT06654752

RECRUITING 400 participants

A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators

Nicole Hamblett

NCT06504589

RECRUITING 400 participants

Natural History of Noncirrhotic Portal Hypertension

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NCT02417740

RECRUITING Phase 1 260 participants

Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary Disease

Mario Castro, MD, MPH

NCT04624490

RECRUITING Phase 1 255 participants

Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated

NCT06154447

RECRUITING NA 240 participants

Exercise in Child Health

University of California, Irvine

NCT05359991

RECRUITING Phase 3 210 participants

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Vertex Pharmaceuticals Incorporated

NCT05422222

RECRUITING 210 participants

Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

Sonya Heltshe

NCT04509050

RECRUITING 200 participants

Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)

Boston Children's Hospital

NCT06683092

RECRUITING Phase 2 192 participants

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

ReCode Therapeutics

NCT06237335

RECRUITING 170 participants

Impact of Sinus Surgery on Individuals With Cystic Fibrosis

University of California, Los Angeles

NCT04469439

RECRUITING 150 participants

Remote Sputum Collection in Cystic Fibrosis

University of Pennsylvania

NCT06950892

RECRUITING 146 participants

Health Outcomes of Parents With Cystic Fibrosis-Aim 2

University of Pittsburgh

NCT06296394

RECRUITING NA 132 participants

Lung Transplant READY CF 2: CARING CF Ancillary RCT

University of Washington

NCT06032273

RECRUITING Phase 1 128 participants

Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants

Vertex Pharmaceuticals Incorporated

NCT07283770

RECRUITING Phase 4 100 participants

Markers of Osteoporosis in Cystic Fibrosis

University of Texas Southwestern Medical Center

NCT03921060

RECRUITING 100 participants

Regional Phenotyping of CF and Non-CF Bronchiectasis

Children's Hospital Medical Center, Cincinnati

NCT04793867

RECRUITING 90 participants

Role of Body Fat Distribution in Metabolic and Pulmonary Decline in Cystic Fibrosis (ORBIT-CF)

Emory University

NCT04002882

RECRUITING 80 participants

Sinus Disease in Young Children With Cystic Fibrosis

University of California, Los Angeles

NCT06191640

RECRUITING Phase 4 75 participants

Estrogen Supplementation and Bone Health in Women With CF

Johns Hopkins University

NCT05704036

RECRUITING Early Phase 1 61 participants

Hormonal Responses to a Mixed Meal in People With Cystic Fibrosis

University of Cincinnati

NCT06163482

RECRUITING NA 60 participants

FEED-Cystic Fibrosis (FEED-CF)

Emory University

NCT05766774

RECRUITING Early Phase 1 60 participants

Regional Monitoring of CF Lung Disease

Children's Hospital Medical Center, Cincinnati

NCT06339593

RECRUITING NA 50 participants

Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials

Children's Hospital Medical Center, Cincinnati

NCT04580368

RECRUITING NA 45 participants

Beta-cell Response to Incretin Hormones in Cystic Fibrosis

University of Pennsylvania

NCT01851694

RECRUITING Phase 4 44 participants

BEGIN Novel ImagiNG Biomarkers

Children's Hospital Medical Center, Cincinnati

NCT05517655

RECRUITING Phase 2 42 participants

Targeting Inflammation With Losartan to Improve Response to Modulator Therapy in Cystic Fibrosis.

University of Kansas Medical Center

NCT06364176

RECRUITING NA 40 participants

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

RECRUITING Phase 1 39 participants

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Vertex Pharmaceuticals Incorporated

NCT05668741

RECRUITING 36 participants

Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis

Massachusetts General Hospital

NCT06216704

RECRUITING 30 participants

Impact of Highly Effective Modulator Therapy on the Cystic Fibrosis Microbiome

Dartmouth-Hitchcock Medical Center

NCT05982795

RECRUITING NA 30 participants

Skin-interfaced Colorimetric Bifluidic Sweat Sensor Device for the Diagnosis of Cystic Fibrosis (CF)

Milton S. Hershey Medical Center

NCT05998629

RECRUITING 30 participants

Continuous Glucose Monitoring and OGTT Screen for Cystic Fibrosis Related Diabetes in Cystic Fibrosis

Medical College of Wisconsin

NCT06560463

RECRUITING 30 participants

Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis

University of North Carolina, Chapel Hill

NCT06057714

RECRUITING Phase 2 30 participants

GLP-1 Agonist Therapy in Cystic Fibrosis-Related Glucose Intolerance

University of Pennsylvania

NCT04731272

RECRUITING Phase 3 25 participants

Assessing Tenapanor as a Treatment of CF-related Constipation.

Massachusetts General Hospital

NCT06810167

RECRUITING Phase 2 24 participants

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis

SpliSense

NCT06429176

RECRUITING Early Phase 1 20 participants

Personalized Theratyping Trial

George Solomon

NCT03587961

RECRUITING Phase 1 15 participants

A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

Spirovant Sciences

NCT06526923

RECRUITING NA 15 participants

The Effects of a Low Glycemic Load Diet on Dysglycemia and Body Composition in Adults With Cystic Fibrosis-Related Diabetes

Rhode Island Hospital

NCT05723445

RECRUITING Phase 1 12 participants

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

Krystal Biotech

NCT05504837

RECRUITING Phase 2 12 participants

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

Arcturus Therapeutics

NCT06747858

ACTIVE NOT RECRUITING 5,000 participants

CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

Nicole Hamblett

NCT03350828

ACTIVE NOT RECRUITING Phase 3 822 participants

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Vertex Pharmaceuticals Incorporated

NCT05444257

ACTIVE NOT RECRUITING 600 participants

Gene Modifiers of Cystic Fibrosis Lung Disease

University of North Carolina, Chapel Hill

NCT00037765

ACTIVE NOT RECRUITING 350 participants

Colorectal Cancer Screening in Cystic Fibrosis

University of Washington, the Collaborative Health Studies Coordinating Center

NCT05362344

ACTIVE NOT RECRUITING 300 participants

Strength and Muscle Related Outcomes for Nutrition and Lung Function in CF

Jaeb Center for Health Research

NCT05639556

ACTIVE NOT RECRUITING 285 participants

Prospective Study of Pregnancy in Women With Cystic Fibrosis

Amalia Magaret

NCT04828382

ACTIVE NOT RECRUITING 180 participants

The PROMISE Pediatric Study 6 to 11 Years Old

Nicole Hamblett

NCT04613128

ACTIVE NOT RECRUITING 154 participants

Genetic Modifiers of Cystic Fibrosis (CF) Liver Disease

University of North Carolina, Chapel Hill

NCT00804583

ACTIVE NOT RECRUITING NA 132 participants

Lung Transplant READY CF 2: A Multi-site RCT

University of Washington

NCT06030206

ACTIVE NOT RECRUITING 100 participants

Host and Bacterial Mechanisms During Cystic Fibrosis Pulmonary Exacerbations

National Jewish Health

NCT04354038

ACTIVE NOT RECRUITING 86 participants

Pulmonary Vascular Disease in CF

Children's Hospital Medical Center, Cincinnati

NCT04549077

ACTIVE NOT RECRUITING Phase 4 64 participants

Hyperpolarized Imaging for New Treatments

Children's Hospital Medical Center, Cincinnati

NCT04259970

ACTIVE NOT RECRUITING 63 participants

CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis

Indiana University

NCT04206436

ACTIVE NOT RECRUITING 60 participants

High Resolution Micro OCT Imaging

University of Alabama at Birmingham

NCT03256773

ACTIVE NOT RECRUITING 38 participants

Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI

Children's Hospital Medical Center, Cincinnati

NCT02848560

ACTIVE NOT RECRUITING Phase 4 26 participants

Non-contrast Lung Perfusion Mapping Applied for New Insights in Cystic Fibrosis

Children's Hospital Medical Center, Cincinnati

NCT04467957

ACTIVE NOT RECRUITING Phase 1 18 participants

Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants

Vertex Pharmaceuticals Incorporated

NCT07349394

ACTIVE NOT RECRUITING Early Phase 1 16 participants

Effect of Losartan in Cystic Fibrosis (CF)-NIH Grant #133240

University of Kansas Medical Center

NCT03435939

ACTIVE NOT RECRUITING NA 13 participants

Mechanisms for Vascular Dysfunction and Exercise Tolerance in CF

Augusta University

NCT02690064

ACTIVE NOT RECRUITING NA 10 participants

Implementation of a Clinical Tool to Improve Waitlist Mortality in Patients With Cystic Fibrosis

The Cleveland Clinic

NCT04687475

ACTIVE NOT RECRUITING Phase 2 8 participants

SGLT2 Inhibitor Therapy in Cystic Fibrosis-related Diabetes

University of Minnesota

NCT06149793

COMPLETED 860 participants

HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy

Indiana University

NCT04798014

COMPLETED Phase 3 517 participants

Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis

Novartis Pharmaceuticals

NCT00388505

COMPLETED Phase 2 510 participants

Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis

Boehringer Ingelheim

NCT00737100

COMPLETED 452 participants

A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)

University of Alabama at Birmingham

NCT02477319

COMPLETED Phase 3 330 participants

MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis

Amgen

NCT01180634

COMPLETED Phase 3 318 participants

Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

Syntara

NCT00630812

COMPLETED Phase 2 312 participants

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

COMPLETED Phase 3 279 participants

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

PTC Therapeutics

NCT02139306

COMPLETED NA 267 participants

Early Intervention in Cystic Fibrosis Exacerbation

Johns Hopkins University

NCT01104402

COMPLETED Phase 3 248 participants

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

COMPLETED Phase 3 238 participants

Study of Ataluren (PTC124™) in Cystic Fibrosis

PTC Therapeutics

NCT00803205

COMPLETED NA 222 participants

Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions

Duke University

NCT03938324

COMPLETED Phase 3 221 participants

OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis

Bonnie Ramsey

NCT02054156

COMPLETED Phase 3 214 participants

Open-Label Phase III Long-Term Safety Trial of Liprotamase

Anthera Pharmaceuticals

NCT00449904

COMPLETED Phase 1 171 participants

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

COMPLETED Phase 3 163 participants

Liprotamase Efficacy Trial in Patients With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Anthera Pharmaceuticals

NCT00449878

COMPLETED NA 150 participants

Saline Hypertonic in Preschoolers

University of Washington, the Collaborative Health Studies Coordinating Center

NCT02378467

COMPLETED 150 participants

Utility of CD64 and TLR2 Assays to Diagnose Acute Pulmonary Exacerbations in Cystic Fibrosis

National Jewish Health

NCT04397809

COMPLETED Phase 2 138 participants

Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation

Nivalis Therapeutics

NCT02589236

COMPLETED Phase 3 125 participants

Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

Vertex Pharmaceuticals Incorporated

NCT01707290

COMPLETED Phase 2 124 participants

A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated

NCT03224351

COMPLETED 123 participants

Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

University of Colorado, Denver

NCT00788359

COMPLETED 120 participants

G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)

Steven M Rowe

NCT01521338

COMPLETED 112 participants

T Cell Memory Fuels the Innate Response in Chronic CF Lung Disease

National Jewish Health

NCT04397861

COMPLETED Phase 2 110 participants

Study of LYM-X-SORB™ to Improve Fatty Acid and Choline Status in Children With CF and PI

Avanti Polar Lipids

NCT00406536

COMPLETED Phase 3 108 participants

Diabetes Therapy to Improve BMI and Lung Function in CF

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NCT00072904

COMPLETED NA 105 participants

Feasibility of a Mobile Medication Plan Application in CF Patient Care

Boston Children's Hospital

NCT03637504

COMPLETED Phase 2 105 participants

Aztreonam for Inhalation (AI) in Patients With Cystic Fibrosis & P. Aeruginosa Infection

Gilead Sciences

NCT01055847

COMPLETED Phase 3 100 participants

Cystic Fibrosis - Insulin Deficiency, Early Action

Sydney Children's Hospitals Network

NCT01100892

COMPLETED 95 participants

An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated

NCT01863238

Phase Distribution

PhaseTrial count
Phase 1 34
Phase 2 27
Phase 3 19
Phase 4 15

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.

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.

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