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Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

Phenylketonuria Clinical Trial

Official title:
A Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With Phenylketonuria

Clinical Trial Summary

The purpose of this study is to assess the safety and tolerability of injections of rAvPAL-PEG in subjects with PKU.

Clinical Trial Description

This is a Phase 1, open-label, single-dose study in approximately 35 subjects with PKU who are 16 to 50 years old. Seven cohorts are planned, each consisting of 5 subjects; the cohorts as planned are 0.001, 0.003, 0.01, 0.03, 0.1, 0.3, and 1.0 mg/kg. Increasing doses of rAvPAL-PEG will be assessed sequentially until the final dose is evaluated or any of the stopping criteria are reached. Subjects will each receive a single dose and then will be followed for a total of 42 days (6 weeks) with visits to the clinical research unit (CRU) as specified in the Schedule of Events Toxicity will be measured according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, v 3). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00634660
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Completed
Phase Phase 1
Start date May 2008
Completion date October 2009
View Details
See also
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