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

About Cystic Fibrosis research: https://www.cff.org/Trials/Pipeline

About clinical trials:  https://www.cff.org/trials/finder

Clinical trials.gov / Cystic Fibrosis: http://clinicaltrials.gov/ct2/results?term=cystic+fibrosis

Actively Enrolling

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

This study lasts for one year and involves visits that start out weekly, then biweekly, then every 4 weeks with phone calls between until study end. Payment and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: 50-90%
  • Class I or II CFTR mutations only

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02677701?term=teach+azithromycin&rank=1

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


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

This study involves 8 visits and phone calls between over about 7 months. Payment and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: 40-100%
  • At least one IV-treated exacerbation in the past year

Principal Investigator: Cesar Trillo-Alvarez, MD

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

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


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 new triple combo therapy) 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 352-294-5195.


TEACH: Testing the effect of adding oral azithromycin to inhaled tobramycin in people with CF

This study involves only 3 short visits with the option to continue open-label for 2 additional visits. Payment and mileage reimbursement provided.

Eligible participants:

  • Ages 12 years and older
  • FEV1: 25-100%
  • at least 2 recent respiratory cultures positive for pseudomonas

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02677701?term=teach+azithromycin&rank=1

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


A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis

This study will look at oral CFTR modulator PTI-808 in combination with PTI-801 with and without PTI-428 vs matching placebo for 28 days. Payment and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and up
  • FEV1 40-90%

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03251092?cond=Cystic+Fibrosis&intr=PTI-808&lead=Proteostasis&phase=0&rank=2

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


STOP2: Standardized Treatment of Pulmonary Exacerbations II

Pulmonary exacerbations are treated with varying antibiotics for varying time periods based on individual needs determined by patients, their families and care providers. This study will look at the safety and effectiveness of three different lengths of IV antibiotic treatment for pulmonary exacerbations. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older

Principal Investigator: Jorge Lascano, MD

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

If you are interested in participating in this study or would like more information, please contact Erin Silverman, PhD at 352-273-5870 or Noni Graham, MPH at 352-294-5195.


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

This study will quantify over a 10 year period the incidence of fibrosing colonopathy in US patients with CF treated with pancreatic enzyme replacement therapy (PERT).

Principal Investigator: Jorge Lascano, MD

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

If you are interested in participating in this study or would like more information, please contact Wanda Nichols, RN at 352-273-5712.


The Role of Diseases in Macrophage Function

Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) share a similar pathway during the folding of the affected protein. In both conditions the mutation leading to the disease causes a misfolding of the affected protein causing several biological effects that we intend to explore. Investigators will collect peripheral blood monocytes, pulmonary function tests and serum C-reactive protein levels from PIMM, PIMZ, PISZ, PIZZ and other rare allele individuals, along with healthy controls and individuals with cystic fibrosis (CF), specifically, homozygous for Delta F508 CFRT mutation, measure the differences in the gene expression of AAT and various other genes in these monocytes at various stages of maturation to macrophages, evaluate the differences in the macrophage function, and determine the mechanisms contributing to these differences.

Principal Investigator: Mark Brantly, MD

Clinicaltrials.gov link:  http://clinicaltrials.gov/ct2/show/NCT01851642?term=alpha-1+antitrypsin+and+macrophage&rank=1

If you are interested in participating in this study or would like more information, please contact Jesse West at 352-273-8666.


Studies Closed to Enrollment

Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose was to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study was comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02279498?term=anthera+solution&rank=1

A randomized, double-blind, parallel group, placebo-controlled study to investigate the optimal dose regimen and efficacy and safety or oral cysteamine treatment in adult CF patients experiencing a CF exacerbation

This 21-day study involves adding cysteamine to the therapy regimen of a patient experiencing an exacerbation.

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03000348?term=cysteamine+cf&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

This 5-year observational study looks at the effectiveness and long-term safety of the TOBI Podhaler and other FDA-approved antipseudomonal drugs.

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


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


EMPIRE-CF: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients with Cystic Fibrosis

This study evaluated the safety and efficacy of CTX-4430 administered once daily for 48 weeks for treatment of CF. CTX-4430 is a novel synthetic, small-molecule, LTA4H inhibitor being developed to address the underlying pulmonary inflammation in CF.

Clinicaltrials.gov link:  https://www.clinicaltrials.gov/ct2/show/NCT02443688


SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin

Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose was to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study was comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02279498?term=anthera+solution&rank=1