Clinical researchers at Nationwide Children's are committed to identifying new approaches for the prevention, diagnosis and treatment of cystic fibrosis, taking research discoveries from the lab to the patient's bedside.
Overview: Cystic-Fibrosis-Related Clinical Trials at Nationwide Children's
Psychosocial Aspects of Living with Cystic Fibrosis
We are participating in a multi-center study examining the psychosocial aspects of living with a chronic disease such as cystic fibrosis. When adolescents ignore thier disease, they are more likely to see rapid advancement of pulmonary damage, wich may lead to a shortening of life. Adolescence is a pivotal time in cystic fibrosis. This project aims to determine what psychosocial factors influence patients' desire and ability to keep up with recommended care and recognize and acknowledge symptoms as a way of helping them make healthy choices.
Effeciveness of Antibiotics to Control Pulmonary Infections
Since pulmonary infections and their related inflammation are the major cause of complications and premature death in cystic fibrosis, we are examining the effectiveness of medications (especially antibiotics) with novel delivery systems being developed for control of pulmonary infections.
Medications to to Remove Thickened Secretions from the Cystic Fibrosis Airway
We are examining the efficacy and tolerance of medications being developed to help remove the thickened secretions from the cystic fibrosis airway. This study is being conducted in infants, which is rare, but offers the promise of intervention at an earlier age with far-reaching potential benefits.
Medication Targeting the Primary Defect in Cells of Cystic Fibrosis Patients
We are examining the safetey and effectiveness of mediations which target the primary defect in cells of cystic fibrosis patients. At present, these agents are directed at defects due to specific genes (there are more than 2,000). These agents may be able to help cystic fibrosis cells function like non-cystic fibrosis airway cells do. These are among the first candidate mediccations targeting the primary defect in cystic fibrosis.
Exercise Capabilities in Cystic Fibrosis Patients
Our doctors are examining exercise capabilities in cystic fibrosis patients to help identify tests/strategies that could help detect when patinet are in the early stages of respiratory compromise, before more obvious dangerous and life-threatening symptoms appear.
Determining Appropriate Endpoints for Cystic Fibrosis Studies
Along with national partners, we are working to determine appropriate endpoints for studies in cystic fibrosis patients. This is incredibly important because it will be how we evaluate the many new potential therapies. This is a major issue, since the advances in overall therapies have led to slowed progression of disease. We are working to determine more appropriate measures such as quality of life and/or CT scans, early life lung functions, days of more versus less symptoms. We may find that clinically undetectable changes in biologic markers are more informative.
Diabetes in Adolescents and Adults with Cystic Fibrosis
We are examining ways to monitor and control and perhaps change the course of the unusal diabetes seen in adolescents and adults with cystic fibrosis. Using a new therapy for diabetes not previously explored in cystic fibrosis, we hope to see benefits that delay the onset of overt diabetes and control the sugar fluctuations seen in diabetes in a less invasive way.