drugset

drugset / Trial / NCT01212744

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

NCT01212744 ↗

Phase 2 Completed 16 enrolled BioMarin Pharmaceutical

NaSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU.

Timeline

Start: 2011-03
Primary completion: 2015-04
Completion: 2015-04

Publications

Burton BK, Longo N, Vockley J, Grange DK, Harding CO, Decker C, Li M, Lau K, Rosen O, Larimore K, Thomas J; PAL-002 and PAL-004 Investigators. Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up. Mol Genet Metab. 2020 Aug;130(4):239-246. doi: 10.1016/j.ymgme.2020.06.006. Epub 2020 Jun 16.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Pegvaliase	Protein / enzyme biologic	0.06 mg/kg	Subcutaneous
Subject	Pegvaliase	Protein / enzyme biologic	0.1 mg/kg	Subcutaneous
Subject	Pegvaliase	Protein / enzyme biologic	0.2 mg/kg	Subcutaneous
Subject	Pegvaliase	Protein / enzyme biologic	0.4 mg/kg	Subcutaneous
Subject	Pegvaliase	Protein / enzyme biologic	0.6 mg/kg	Subcutaneous
Subject	Pegvaliase	Protein / enzyme biologic	0.8 mg/kg	Subcutaneous

Indications