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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, Wanda Nichols, RN cfcenter@medicine.ufl.edu

About Cystic Fibrosis research: www.cff.org/treatments/Pipeline

About clinical trials:  www.cff.org/research/clinicalresearch

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

(Pill That Hits A Cause Of Cystic Fibrosis Beats Expectations) http://www.npr.org/blogs/health/2011/02/24/133995182/pill-that-hits-a-cause-of-cystic-fibrosis-beats-expectations?sc=emaf

Actively Enrolling:

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).

Principal Investigator:  Jorge Lascano, MD

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

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 to address the underlying pulmonary inflammation in CF.

Principal Investigator:  Jorge Lascano, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02443688?term=celtaxsys+empire&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 Noni Graham, MPH at 352-294-5195.

Closed to Enrollment:

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).

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02343445?term=PARION+CLEAN-CF&rank=1

If you would like more information, please contact Noni Graham, MPH at 352-294-5195.

Closed:

AZLI CAT:  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy (CAT) Regimen of Inhaled Antibiotics for the Treatment of Chronic Pulmonary Pseudomonas aeruginosa Infection in Subjects with Cystic Fibrosis. 

This study will evaluate the safety and efficacy of continuous alternating therapy with AZLI and tobtamycin inhalation solution (TIS) in adult & pediatric subjects with cystic fibrosis (CF) and pulmonary Pseudomonas aeruginosa (PA) infection.

Principal Investigator:  Jorge Lascano, MD

Clinicaltrials.gov link:  http://clinicaltrials.gov/ct2/show/NCT01641822?term=aztreonam+and+cystic+fibrosis&rank=11

If you are interested in participating in this study or would like more information, please contact Wanda Nichols, RN at 352-273-5712 or Jessica Sanders, CPT at 352-273-6339.

Closed:

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.

Principal Investigator:  Jorge Lascano, MD

Clinicaltrials.gov link: http://clinicaltrials.gov/ct2/show/NCT01695343?term=kb-001&rank=1

If you would like more information, please contact Wanda Nichols, RN at 352-273-5712

Closed:

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.

Principal Investigator: Jorge Lascano, MD

Clinicaltrials.gov link: http://clinicaltrials.gov/ct2/show/NCT02056132?term=exhaled+breath+condensate+and+cystic+fibrosis&rank=2

If you would like more information, please contact Wanda Nichols, RN at 352-273-5712

Closed:

Vertex:  A Phase 3, Randomized, Double-Blind,Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Homozygous for the F508del-CFTR Mutation.  Vertex Study Number: VX12-809-103

The purpose of this study is to evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in persons 12 years and older with Cystic Fibrosis who are homozygous for the F508del mutation.

Principal Investigator:  Jorge Lascano, MD

Clinicaltrials.gov link:  http://clinicaltrials.gov/ct2/show/study/NCT01807923?term=lumacaftor+and+ivacaftor&rank=5&show_locs=Y#locn

If you would like any more information on this study, please contact Wanda Nichols, RN at 352-273-5712.