Table 1.

Clinical trials of oral sapropterin

Disease state	Intervention	Duration, wk	Results	Clinical trial ID
Systemic hypertension	Oral sapropterin, 5 mg/kg, BID	8	No statistically significant effect on SBP vs placebo (http://www.bmrn.com/)	NCT00325962
Pulmonary arterial hypertension	Oral sapropterin, dose escalation every 2 wks from 2.5 to 5 to 10 mg/kg/d + 2 d of 20 mg/kg BID	6	No statistically significant effects vs placebo (http://www.bmrn.com/)	NCT00435331
Sickle cell disease	Oral sapropterin, dose escalation every 4 wks from 2.5 to 5 to 10 to 20 mg/kg/d	16	Improvement of RH-PAT at 8, 12, and 16 wks of treatment. Sapropterin was well tolerated (http://www.bmrn.com/)	NCT00445978
Coronary artery disease	400 mg/d or 700 mg/d	2–6	No statistically significant difference in clinical end points or in dilation or NOS coupling in ex vivo vessels	NCT00423280, see reference [96••]
Peripheral arterial disease	400 mg BID	24	Not reported to date (Start date: Dec 2006; est. completion: Jan 2009)	NCT00403494
Systolic hypertension	Oral sapropterin, 5 mg/kg, BID	8	Not reported (Start date: Dec 2008)	NCT00802893
Chronic kidney disease	Oral sapropterin, 400 mg BID + 400 mg vitamin C BID for the second 6 wks	12	Not reported to date (Start date: May 2008; est. completion: Aug 2009)	NCT00625820
All-cause endothelial dysfunction	Oral sapropterin, 5 mg/kg oral ± 500 mg vitamin C BID	2	Not reported to date (Start date: Sept 2007; est. completion: March 2009)	NCT00532844
Liver cirrhosis, portal hypertension	Oral sapropterin, dose escalation weekly from 5 to 10 mg/kg/d	2	Ongoing (Start date: Oct 2011; est. completion: Jan 2013)	NCT01456286

Synthetic 6R-BH4, Trade name: Kuvan (manufactured by Biomarin Pharmaceutical Inc., Novato, CA)

BID twice daily; SBP systolic blood pressure; RH-PAT reactive hyperemia-peripheral arterial tonometry (a measurement of vasodilation following temporary constriction of the forearm); NOS nitric oxide synthase; est. estimated.