Ignite Creation Date:
2024-05-05 @ 5:19 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00435331
Status:
COMPLETED
Last Update Posted:
2013-06-19
First Post:
2007-02-12
Brief Title:
6R-BH4 Pulmonary Arterial Hypertension Study
Sponsor:
Vanderbilt University
Organization:
Vanderbilt University
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2013-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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 Description:
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
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None