Safety and Tolerability Study of Single and Multiple PL54 Injections in Adults with Phenylketonuria Aged 18 to 55

What this Trial Is About

Researchers are evaluating the safety and tolerability of single and multiple doses of PL54 injections in adults aged 18 to 55 years with phenylketonuria PKU. This Phase I, open-label, dose-escalation study aims to compare the safety profiles between single and multiple dosing regimens and assess pharmacokinetics, pharmacodynamics, and immunogenicity of the drug. The study consists of two phases Phase Ia involves a single subcutaneous injection of PL54 at increasing doses from 0.01 mgkg to 0.5 mgkg, followed by a 28-day observation period with several clinic visits. Phase Ib includes multiple weekly subcutaneous injections for four weeks at doses from 0.04 mgkg to 0.25 mgkg, with a 35-day follow-up after the last dose and scheduled assessments. Dose escalation depends on safety data and committee review. Participants will attend multiple clinic visits for drug administration and monitoring, including assessments of adverse events and blood phenylalanine levels. Researchers will record the severity and relationship of any side effects and measure changes in blood phenylalanine concentration from baseline. The total participation duration is approximately six weeks for Phase Ia and up to nine weeks for Phase Ib, with safety and laboratory assessments throughout.

A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)