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

The primary objective of this clinical trial is to evaluate the safety and tolerability of single and multiple administrations of PL54 in patients aged 18-55 years. The key questions it aims to answer include: How safe and tolerable is PL54 in PKU patients following single and multiple administrations? Researchers will compare the safety and tolerability profiles between single and multiple dosing regimens to assess PL54. Participants will be required to: Phase Ia (Single Administration): Receive a single subcutaneous injection of PL54 based on body weight. Undergo a 28-day observation period post-injection. Visit the clinic for assessments on: D1 (administration day), D2, D7, D8, D10, D15, D22, and D29. Phase Ib (Multiple Administrations): Receive subcutaneous injections of PL54 every 7 days (4 doses total) based on body weight. Undergo a 35-day observation period after the last injection. Visit the clinic for assessments on: D1 (first administration), D3, D8, D15, D21, D22, D29, D36, D43, and D57 Data to be recorded include: Incidence, severity, seriousness, relationship to PL54 treatment, duration, and outcome of adverse events (AEs). Change from baseline in blood phenylalanine (Phe) concentration.

NCT Number	NCT07672756
Lead Sponsor	Chongqing Peg-Bio Biopharm Co., Ltd.
Conditions	Phenylketonuria (PKU)
Enrollment	48 participants
Start Date	2025-07-20
Primary Completion	2027-06-01 (estimated)
Study Completion	2027-08-01 (estimated)
Updated on ClinicalTrials.gov	2026-07-01