[Translation] A multicenter, open-label, dose-escalation, phase I clinical study evaluating PL54 injection in adult patients with phenylketonuria (PKU)
主要目的:评估PL54多次皮下注射在成年PKU患者中的安全性和耐受性。
次要目的:
(1) 评估PL54多次皮下注射在成年PKU患者中的PK特征;
(2) 评估PL54多次皮下注射在成年PKU患者中的PD特征;
(3) 评估PL54多次皮下注射在成年PKU患者中的免疫原性;
(4) 结合安全性、免疫原性及目标PD[血液中苯丙氨酸(Phe)浓度:60 ~ 600 μmol/L]初步探索合适的给药方法。
[Translation] Primary objective: To evaluate the safety and tolerability of multiple subcutaneous injections of PL54 in adult PKU patients.
Secondary objectives:
(1) To evaluate the PK characteristics of multiple subcutaneous injections of PL54 in adult PKU patients;
(2) To evaluate the PD characteristics of multiple subcutaneous injections of PL54 in adult PKU patients;
(3) To evaluate the immunogenicity of multiple subcutaneous injections of PL54 in adult PKU patients;
(4) To preliminarily explore the appropriate administration method based on safety, immunogenicity and target PD [blood phenylalanine (Phe) concentration: 60 ~ 600 μmol/L].