Clinical Trial: 6R-BH4 Pulmonary Arterial Hypertension Study
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase 1, Multicenter, Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of 6R-BH4 in Subjects With Pulmonary Arterial Hypertension
Brief Summary:
The purpose of this study is to determine whether the addition of sapropterin dihydrochloride (6R-BH4) to existing treatment has any effect in patients with pulmonary arterial hypertension (PAH). Patients with PAH have low levels of a substance called nitric oxide (NO). Tetrahydrobiopterin (BH4) is a substance produced by the body that is an essential requirement in the formation of NO. NO is thought to be helpful in keeping blood vessels in the lung healthy. 6R-BH4 is an experimental (unproven) medicine made in the lab that is very much like the BH4 that our own body makes. The researchers are investigating whether 6R-BH4 can be added safely to current treatment for PAH and whether there is any evidence of benefit from its use. The study will take approximately one year to complete from the time recruitment begins.
The primary objective of the study is to evaluate the safety of oral 6R-BH4, administered in escalating doses in addition to standard care, in subjects with pulmonary arterial hypertension (PAH).
The secondary objective of the study is to evaluate change in biochemical markers of endothelial dysfunction and nitric oxide synthetase activity (coupled and uncoupled) in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.
The third objective of the study is to evaluate change in biomarkers of disease progression, 6-minute walk (6MW) distance, Borg dyspnea scores, and quality of life (QOL) measures in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.
Detailed Summary:
The primary objective of the study is to evaluate the safety of oral 6R-BH4, administered in escalating doses in addition to standard care, in subjects with pulmonary arterial hypertension (PAH).
The secondary objective of the study is to evaluate change in biochemical markers of endothelial dysfunction and nitric oxide synthetase activity (coupled and uncoupled) in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.
The third objective of the study is to evaluate change in biomarkers of disease progression, 6-minute walk (6MW) distance, Borg dyspnea scores, and quality of life (QOL) measures in subjects with PAH receiving escalating doses of oral 6R-BH4 in addition to standard care.
Sponsor: Vanderbilt University
Current Primary Outcome: To evaluate the safety of oral 6R-BH4, administered in escalating doses in addition to standard care, in subjects with pulmonary arterial hypertension (PAH). [ Time Frame: Up to 14 weeks ]
Original Primary Outcome: To evaluate the safety of oral 6R-BH4, administered in escalating doses in addition to standard care, in subjects with pulmonary arterial hypertension (PAH).
Current Secondary Outcome:
- Change in biochemical markers of endothelial dysfunction and nitric oxide synthetase activity (coupled and uncoupled). [ Time Frame: Up to 14 weeks ]
- Change in biomarkers of disease progression, 6-minute walk (6MW) distance, Borg dyspnea scores, and quality of life (QOL) measures. [ Time Frame: Up to 14 weeks ]
Original Secondary Outcome: To evaluate change in biochemical markers of endothelial dysfunction and nitric oxide synthetase activity (coupled and uncoupled) in subjects with PAH receiving escalating doses of 6R-BH4 in addition to standard care
Information By: Vanderbilt University
Dates:
Date Received: February 12, 2007
Date Started: March 2008
Date Completion:
Last Updated: June 17, 2013
Last Verified: June 2013
