NCT00924703

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKU

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 16 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.

Detailed description

PAL-003 is designed to evaluate long-term treatment of subjects who are continuing to take rAvPAL-PEG. Subjects'previous rAvPAL-PEG dosing will continue in PAL-003. In PAL-003, each subject's dose will be adjusted as needed to attain or maintain blood Phe concentrations of 60-600 µmol/L. rAvPAL-PEG dose will be based on either a subject's weight or will be a fixed dose (subjects who have maintained blood Phe levels to 60-600 µmol/L for at least 2 consecutive weeks and who have maintained a stable rAvPAL-PEG dose for at least 2 consecutive weeks). Doses will be evaluated on an individual basis.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	rAvPAL-PEG	The doses are planned to be in the same range as those tested in PAL-002 or PAL-004 and then modified either by increasing or decreasing the dose, adhering to an upper limit up to 5.0 mg/kg per week or 375 mg/week, considering each subject's individual responses related to safety and efficacy.

Timeline

• Start date: 2010-01-13
• Primary completion: 2019-01-31
• Completion: 2019-01-31
• First posted: 2009-06-19
• Last updated: 2021-10-12
• Results posted: 2021-10-12

Locations

14 sites across 1 country: United States