Status: Active, not recruiting
IRB Number: 00056411
Specialty: Pediatric Genetics
This study will be a Phase 2, multi-center, open-label, dose-finding study to evaluate safety, efficacy and tolerability of rAvPAL-PEG in patients with PKU for a treatment period of 24 weeks. Approximately 24 rAvPAL-PEG-naïve eligible patients with PKU will be enrolled in the study. The total study duration for an individual patient will be up to 29 weeks: up to 4 weeks screening, 24 weeks of treatment with rAvPAL-PEG, and 1 week for post-treatment safety follow-up. Upon completion of the study, all patients will be offered to enroll in the long-term extension study PAL-003.
The primary objective of the study is:
• To identify a safe and efficacious dosing regimen of rAvPAL-PEG by evaluating multiple schedules involving bolus, induction, titration, and maintenance dosing.
The secondary objectives of the study are:
• To evaluate immune response of multiple SC doses of rAvPAL-PEG on reduction of blood Phe concentrations in patients with PKU.
• To evaluate pharmacokinetic (PK) profile of multiple SC doses of rAvPAL-PEG on reduction of blood Phe concentrations in patients with PKU.
The exploratory objectives of the study are as follows:
• To assess the relationship between change in blood Phe concentration and diet following administration with rAvPAL-PEG in patients with PKU.
• To assess the relationship of change in metabolic panel from baseline to end of study following administration of rAvPAL-PEG in patients with PKU.
Principal Investigator: Nicola Longo
Department: Pediatric Genetics
Co Investigator: Dave Viskochil
Co Investigator: Ashley Warnock
Co Investigator: Lorenzo Botto