Cystic Fibrosis (CF)

Research is an important part of Cystic Fibrosis (CF) care because it is the only way we can develop better treatments and, hopefully, a cure for CF in the near future. We have several clinical research opportunities. These trials involve volunteers who help scientists answer specific questions about a new treatment or a new way of using an old treatment.

For more information on our ongoing clinical research, please contact our Clinical Research Coordinator, Noni Graham, MPH at noni.graham@medicine.ufl.edu or 352-294-5195


Actively Enrolling

Anti-Infective Therapies


A Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of the safety and pharmacokinetics of AR-501 (gallium citrate), administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis subjects

This study is being done to determine the safety of weekly dosing of inhaled AR-501 in people with CF. Payment provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: At least 45%
  • Chronic lung infection with pseudomonas aeruginosa

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03669614?term=ar-501&cond=Cystic+Fibrosis&draw=2&rank=1

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at noni.graham@medicine.ufl.edu or 352-294-5195


CX-280-202: A Phase 1B randomized, double-blind, placebo-controlled trial to evaluate the safety of CB-280 in patients with cystic fibrosis

This study is being done to determine the safety and tolerability of oral CB-280. Payment provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: 40-90%
  • Chronic lung infection with pseudomonas aeruginosa

Learn more here: https://mediasite.video.ufl.edu/Mediasite/Play/554372cc8fee4484a111f5c833c8ca001d 

Principal Investigator: Cesar Trillo-Alvarez, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT04279769?term=cb-280&cond=Cystic+Fibrosis&draw=2&rank=1

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at noni.graham@medicine.ufl.edu or 352-294-5195


Observational Studies


Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis

This 2-part study aims to improve diagnosis of NTM disease and evaluate a specific algorithm used for treatment of NTM disease. Payment provided.

Eligible participants:

  • Ages 6 years and older with a recent NTM positive sputum culture
  • Enrolled in the CF Patient Registry
  • Can provide sputum samples
  • NOTE: Only those who’ve participated in the PREDICT portion of the study are eligible to be evaluated for participation in PATIENCE

Principal Investigator: Silvia Delgado Villalta, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02073409?term=ntm&cond=Cystic+Fibrosis&draw=2&rank=2

If you are interested in participating in this study or would like more information, please contact Sarah Barbey, MS at 352-273-5085.


THE CHEC-SC Cohort Study: Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes

This study involves only one visit with option to re-enroll if participant changes CFTR modulator. Payment provided.

Eligible participants:

  • Ages 4 months and older
  • Enrolled in the CF Patient Registry
  • Prescribed a CFTR Modulator (e.g., Kalydeco, Orkambi, Symdeko or Trikafta) for at least 3 months

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT03350828?term=NCT03350828&rank=1

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at noni.graham@medicine.ufl.edu or 352-294-5195


Other


A master protocol to test the impact of discontinuing chronic therapies in people with cystic fibrosis on highly effective CFTR modulator therapy (SIMPLIFY)

This study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline or Pulmozyme in people who take Trikafta. Payment provided.

Eligible participants:

  • Ages 12 years and older
  • Enrolled in the CF Patient Registry
  • Prescribed Trikafta for at least 3 months

Principal Investigator: Silvia Delgado Villalta, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04378153?term=SIMPLIFY&cond=Cystic+Fibrosis&draw=2&rank=1

If you are interested in participating in this study or would like more information, please contact Alexa Smith, BAS at 352-273-7505.


Staff


Closed to Enrollment


Therapies to Restore CFTR Function


A Phase 1/2, Randomized, Double-Blind, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 (CO-hCFTR mRNA/ICE LNP) Administered by Nebulization to Adult Subjects with Cystic Fibrosis: RESTORE-CF Study

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT03375047?term=mrt5005&cond=Cystic+Fibrosis&draw=2&rank=1


A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects with Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT03525574?term=vx17-445-105&cond=Cystic+Fibrosis&draw=2&rank=1


Anti-Inflammatory Therapy


APPLAUD: A Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Efficacy and Safety of LAU-7B in the treatment of Cystic Fibrosis in Adults

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT03265288?term=Laurent&cond=Cystic+Fibrosis


Anti-Infective Therapy


IGNITE: A Phase 2, multicenter, randomized, placebo-controlled study of IV gallium nitrate in patients with CF

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02354859


STOP2: Standardized Treatment of Pulmonary Exacerbations II

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02781610?term=stop+cystic+fibrosis&rank=2


Observational Studies


A prospective study to evaluate the biological and clinical effects of significantly corrected CFTR function (the PROMISE Study)

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04038047?term=promise&cond=Cystic+Fibrosis&draw=2&rank=1


A Phase 4 prospective observational study in CF patients with chronic pseudomonas aeruginosa and treated with TOBI Podhaler or other FDA approved inhaled antipseudomonal antibacterial drugs

Clinicaltrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT02449031


CFFC:  A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol across Sponsors

Clinicaltrials.gov link:  http://clinicaltrials.gov/ct2/show/NCT01652157?term=fibrosing+colonopathy+and+cystic+fibrosis&rank=1