drugset / Trial / NCT01772758

Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

NCT01772758

Phase 2 Completed 64 enrolled Augusta University
Non-randomizedParallel-groupDouble-blindOther

Summary

Perhaps one of the most disturbing aspects of Cystic Fibrosis (CF) is the associated premature death. Oxidative stress has been observed in patients with CF and exercise intolerance has been shown to predict mortality in patients with CF, regardless of how healthy their lungs are. A critical barrier to improving the quality of life and longevity in patients with CF is our lack of knowledge regarding the different reasons why patients with CF cannot exercise to the level of their peers. We have collected preliminary data to support our central hypothesis that oxidative stress contributes to the impairment in blood vessel function at rest and during exercise which ultimately oxygen transport and delivery resulting in exercise intolerance. Exercise is therapeutic medicine for patients with CF and this investigation represents a major breakthrough in the approach to begin understanding the physiological mechanisms which contribute to exercise intolerance in these patients.

Timeline

Start
2011-08
Primary completion
2016-06-21
Completion
2016-06-21

Drugs

EvaluationDrugModalityDoseRoute
Subject sapropterin Unknown 5 mg/kg Oral
Subject sapropterin Unknown 20 mg/kg Oral

Indications