Pulmonary Arterial Hypertension Clinical Trial
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
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.
;
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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