Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

Phase 3 Completed 111 enrolled BioMarin Pharmaceutical

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.

Timeline

Start: 2006-07
Primary completion: 2009-08
Completion: 2009-08

Publications

Burton BK, Nowacka M, Hennermann JB, Lipson M, Grange DK, Chakrapani A, Trefz F, Dorenbaum A, Imperiale M, Kim SS, Fernhoff PM. Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study. Mol Genet Metab. 2011 Aug;103(4):315-22. doi: 10.1016/j.ymgme.2011.03.020. Epub 2011 Mar 31.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	sapropterin	Unknown	5 mg/kg	Oral
Subject	sapropterin	Unknown	20 mg/kg	Oral

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

Summary

Timeline

Publications

Drugs

Indications