In 2002, Cystic Fibrosis Foundation Therapeutics, Inc. the nonprofit drug development affiliate of the Cystic Fibrosis Foundation, selected Nationwide Children’s Cystic Fibrosis Care Center to be part of the Therapeutics Development Network (TDN). Nationwide Children’s is one of 16 TDN centers in the United States. These centers are accredited to conduct clinical trials of possible new treatments for cystic fibrosis.
In 2009, Nationwide Children’s was selected to serve as one of 13 translational research centers for the Cystic Fibrosis Foundation’s Therapeutics Development Network. These translational research centers lead the newest Phase I clinical trials and provide scientific direction to the entire network.
Right Ventricular Function in Healthy Cystic Fibrosis Patients
This study aimed to compare the right ventricular function between healthy cystic fibrosis (CF) patients and non-CF patients to determine whether differences exist. While differences were seen, further studies are needed to determine the significance of these findings for the pediatric CF population.
Access an abstract of this study: Decreased Right Ventricular Function in Healthy Pediatric Cystic Fibrosis Patients Versus Non-Cystic Fibrosis Patients. Pediatr Cardiol. 2012 Jun 10. [Epub ahead of print]
Using CT Lung Images to Assess the Airway of Children with Cystic Fibrosis
This study discusses computer-assisted methods for assessing airway and vessel dimensions in infants and children with cystic fibrosis, using CT lung images acquired at low pediatric radiation doses.
Access an abstract of this study: Image Analysis for Cystic Fibrosis: Computer-Assisted Airway Wall and Vessel Measurements from Low-Dose, Limited Scan Lung CT Images. J Digit Imaging. 2012 May 2. [Epub ahead of print]
How White Blood Cells React to Bacterial Infection in Cystic Fibrosis
The drug-resistant bacterium, B. cenocepacia, can worsen inflammation in cystic fibrosis. This study examines how macrophages (large white blood cells) respond to the bacterium in cystic fibrosis patients and suggests that corticosteroids might suppress the resulting cytokine responses.
Access an abstract of this study: Exaggerated inflammatory responses mediated by burkholderia cenocepacia in human macrophages derived from cystic fibrosis. Biochem Biophys Res Commun. 2012 Jun 20. [Epub ahead of print]
Gastroesophageal Reflux in Cystic Fibrosis
This review article discusses what is currently known about the mechanisms and management of gastroesophageal reflux in patients with cystic fibrosis.
Access an abstract of this study: Gastroesophageal Reflux in Cystic Fibrosis: Current Understandings of Mechanisms and Management. Curr Gastroenterol Rep. 2012 Apr 13. [Epub ahead of print]
Inpatient Healthcare Trends Among Adult Cystic Fibrosis Patients in the U.S.
Adult cystic fibrosis (CF) patients are an expanding cohort that is taken care of in a variety of hospital settings including adult centers located within pediatric institutions. This study compared costs and discharge rates among adult CF patient hospitalizations in terms of location of hospitalization.
Access an abstract of this study: Inpatient healthcare trends among adult cystic fibrosis patients in the U.S. Pediatr Pulmonol. 2011 Sep 7. doi: 10.1002/ppul.21535. [Epub ahead of print]
Possible Advantages of Conducting Bronchoalveolar Lavages During Sinus Surgeries in CF Patients, Cystic Fibrosis Foundation Therapeutics (Stephen Kirkby)
Infant Study of Inhaled Saline in Cystic Fibrosis – Isis002, University of North Carolina and CF Therapeutics Development Network (Karen McCoy)
A Phase 2, multi-center, randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ΔF508-CFTR Gene Mutation, Vertex Pharmaceuticals, Inc. (Karen McCoy)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects with Cystic Fibrosis and the G551D Mutation, Vertex Pharmaceutical Inc. (Karen McCoy)
2 year study to assess the natural hx of progressive CF lung disease utilizing CT imaging, PFT’s and clinical scores, CF Therapeutics Development Network/Novartis (Karen McCoy)
Cystic Fibrosis Center, CFFT (Karen McCoy)
CF Therapeutic Development Network - Translational TDC Center, CF Therapeutics Development Network (Karen McCoy)
Prevention of Cystic Fibrosis Diabetes, Emory University/Food and Drug Administration
Phase 3b Randomized, Double-Blind, Placebo-Controlled Two-Part Trial to Assess the Safety and Efficacy of Continuous Aztreonam for Inhalation Solution (AZLI) in Subjects with Cystic Fibrosis (CF) and Chronic Burkholderia Species Infection, Gilead Sciences, Inc. (Karen McCoy)
An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX770 in Subjects with Cystic Fibrosis, Vertex Pharmaceuticals (Karen McCoy)
Phase 3 b, Randomized, Double-Blind, Placebo-Controlled Two-Part Trial to Assess the Safety and Efficacy of Continuous Aztreonam for Inhalation Solution (AZLI) in Subjects with Cystic Fibrosis (CF) and Chronic Burkholderia Species Infection, Gilead Sciences, Inc. (Karen McCoy)
Randomized, Placebo-controlled, Double-Blind, Multi-Center Study to Assess the Efficacy, Safety and Tolerability of Study Drug in Cystic Fibrosis Patients with Chronic Infection Due to Pseudomonas Aeruginosa, Insmed Inc. (Karen McCoy)
Prevention of Cystic Fibrosis Diabetes, Emory University (Karen McCoy)
CFTR regulation of caspase-1 and inflammatory cytokines during Burkholderia cepacia infection, The Research Institute at Nationwide Children’s Hospital (Benjamin Kopp)
A phase 3, multi-center multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of MP-376 in stable cystic fibrosis patients, Mpex Pharmaceuticals (Karen McCoy)
Pulmonary vascular hemodynamic in patients with cystic fibrosis, Children’s Hospital Medical Center Cincinnati (Karen McCoy)
Changes in lung structure and function in children with cystic fibrosis, Cystic Fibrosis Foundation Therapeutics (Robert Castile)
iCARE: I change adherence and raise expectations, University of Miami (Karen McCoy)
A Prospective, 5-year Registry Study to Monitor the Susceptibility to Study Drug of PA Isolates from Patients with Cystic Fibrosis in the United States, Anonymous (Karen McCoy)
ALPINE-Open-Label Phase 2 Trial to Evaluate the Safety and Efficacy of Study Drug in Patients with Cystic Fibrosis, Anonymous (Karen McCoy)