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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. There are two types of research: Clinical trials, which involve people living with CF who volunteer to help scientists answer specific questions about a new treatment or a new way of using an old treatment; and medication trials, which test a specific use for a specific medication.

For more information on our ongoing clinical research studies, 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

ARTICLE: Potential Cystic Fibrosis Therapy Gets FDA Fast Track Approval, Clinical Tests to Follow

Actively Enrolling:

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. Stipend and mileage reimbursement provided.

Eligible participants:

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

Principal InvestigatorJorge 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.

Actively Enrolling:

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

The total length of participation for this study is 2 months, and involves a 5-day inpatient infusion and 3 follow-up visits. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1 >25%
  • at least 2 recent respiratory cultures positive for pseudomonas

Principal Investigator: Jorge Lascano, MD

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

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at 352.294.5195.

Actively Enrolling:

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 will look at the effectiveness and long-term safety of the TOBI Podhaler and other FDA-approved antipseudomonal drugs.

Eligible participants:

  • Ages 6 years and older
  • FEV1: 25-80%
  • at least 2 recent respiratory cultures positive for pseudomonas

Principal Investigator: Jorge Lascano, MD

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

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at 352.294.5195.

Actively Enrolling:

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 will evaluate 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 being developed to address the underlying pulmonary inflammation in CF. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18-30
  • FEV1: ≥50%
  • At least one pulmonary exacerbation in the past 12 months

Principal Investigator: Jorge Lascano, MD

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

If you are interested in participating in this study or would like more information, please contact Noni Graham, MPH at 352.294.5195.

Actively Enrolling:

VX15-371-101: A Phase 2a Randomized, Double-blind placebo-controlled, incomplete block, crossover, multicenter, study in subjects ≥12 years of age with CF who are homozygous for the F508del-CFTR mutation and who are being treated with Orkambi.

This study will look at the safety and effectiveness of the inhaled drug VX-371 (formerly P-1037) in saline compared to saline alone. Total length of participation is about 4 months. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 12 years and older
  • Two copies of F508del
  • FEV1: 40-90%

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link:  https://clinicaltrials.gov/ct2/show/NCT02709109?term=VX-371&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.

Actively Enrolling:

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. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: >30%
  • Weighing more than 88 pounds

Principal Investigator: Jorge Lascano, MD

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

Actively Enrolling:

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.

Actively Enrolling:

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, RN at 352.273.8666.

Actively Enrolling:

Psychometric Evaluation of the Gastrointestinal (GI) Symptom Tracker in Patients with Cystic Fibrosis

This study will look at a new tracker in adolescent and adult patients with CF. Only 1-2 brief visits to answer questions about CF, GI symptoms and enzyme replacement therapy. A stipend will be provided.

Eligible participants:

  • Ages 14 years and older
  • Confirmed pancreatic insufficiency for at least 6 months

If you are interested in participating in this study or would like more information, please contact Dawn Baker, ARNP at 352.273.5417 or Ticea Cannon, LPN at 352.294.8489.

Stay tuned for the following new trials:

A Phase I/II, Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis

This study will look at oral PTI-428 as a CFTR amplifier. This trial includes a variety of cohorts, with lengths of participation ranging from 14 to 35 days. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18-55 years
  • FEV1 40-90%

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02718495?term=PTI-428&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.


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.


Gilead: A Phase 2b, Dose-Ranging Study of the Effect of GS-5745 on FEV1 in Adult Subjects with Cystic Fibrosis

This two-part study will look at the effectiveness of the anti-inflammatory drug GS-5745 via subcutaneous injections in adults with CF. Stipend and mileage reimbursement provided.

Eligible participants:

  • Ages 18 years and older
  • FEV1: 40-80%

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT02759562?term=GS-US-404-1808&rank=1

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.

Closed to Enrollment:

VX14-661-110: A Phase 3, Open-label, Rollover study to evaluate the safety and efficacy of long-term treatment with VX-661 in combination with ivacaftor in subjects aged 12 years and older with CF, homozygous or heterozygous for the F508-CFTR mutation

This study is open to subjects previously enrolled in the VX661 group of studies i.e., 103, 106,107,108, 109, 111.


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 of the present study is 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 will be comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).


CLEAN-CF: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of P-1037 Solution for Inhalation in Patients with Cystic Fibrosis

The goal of the study was to evaluate the safety and tolerability of P-1037 and to determine whether the combination of P-1037 with hypertonic saline or P-1037 alone had a greater effect on lung function in patients with CF than placebo (0.17% saline).


KaloBios: A Phase 2, Randomized, Double-blind, Placebo-controlled. Repeat-dose Study of KB001-in Subjects with Cystic Fibrosis Infected with Pseudomonas Aeruginosa

This study evaluated the effect of KB001-A on time-to-need for antibiotic (ABX) treatment for worsening of respiratory tract signs and symptoms.


Exhaled Breath Condensate (EBC) for evaluation of lung infections and exacerbations in patients with Cystic Fibrosis

The purpose of this study was to evaluate possible markers of inflammation that can assist in a noninvasive way in the determination of CF exacerbations.