Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Summary

The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.

Timeline

Start

2005-03

Primary completion

—

Completion

2006-02

Outcome

Met primary endpoint

registry analysis (superiority or other test); Sapropterin Dihydrochloride vs Placebo; p <0.001; ANCOVA NCT00104247 ↗

release “placebo-controlled clinical study of Phenoptin (commonly referred to as 6R-BH4, or BH4) in patients with elevated blood phenylalanine (Phe) levels demonstrated a statistically significant reduction” biomarin.com ↗

release “Results confirm that all pre-specified primary and secondary endpoints were met” biomarin.com ↗

Publications

• Levy HL, Milanowski A, Chakrapani A, Cleary M, Lee P, Trefz FK, Whitley CB, Feillet F, Feigenbaum AS, Bebchuk JD, Christ-Schmidt H, Dorenbaum A; Sapropterin Research Group. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. Lancet. 2007 Aug 11;370(9586):504-10. doi: 10.1016/S0140-6736(07)61234-3.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	sapropterin	Unknown	10 mg/kg	Oral

Indications