NCT00634660

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 16 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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).

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	rAvPAL-PEG	rAvPAL-PEG will be administered as a single, SC injection at dose levels of 0.001, 0.003, 0.01, 0.03, 0.1, 0.3, and 1.0 mg/kg. The duration of treatment is a single dose of study drug with 42 days (6 weeks) of follow-up.

Timeline

• Start date: 2008-05-01
• Primary completion: 2009-04-01
• Completion: 2009-10-01
• First posted: 2008-03-13
• Last updated: 2017-02-01

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study