drugset / Trial / NCT00925054
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
Non-randomizedSequentialOpen-labelTreatment
Summary
The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether repeated administration is safe.
Timeline
- Start
- 2009-09
- Primary completion
- 2015-06
- Completion
- 2015-06
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.
- Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S. Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial. Lancet. 2014 Jul 5;384(9937):37-44. doi: 10.1016/S0140-6736(13)61841-3. Epub 2014 Apr 14.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Pegvaliase | Protein / enzyme biologic | 0.001 mg/kg | Subcutaneous |
| Subject | Pegvaliase | Protein / enzyme biologic | 0.003 mg/kg | Subcutaneous |
| Subject | Pegvaliase | Protein / enzyme biologic | 0.01 mg/kg | Subcutaneous |
| Subject | Pegvaliase | Protein / enzyme biologic | 0.03 mg/kg | Subcutaneous |
| Subject | Pegvaliase | Protein / enzyme biologic | 0.1 mg/kg | Subcutaneous |