A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylke… (NCT07672756) | Clinical Trial Compass
RecruitingPhase 1
A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
China48 participantsStarted 2025-07-20
Plain-language summary
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.
Who can participate
Age range
18 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key Inclusion Criteria:
* Adult patients aged 18 to 55 years, inclusive, regardless of sex.
* Subjects with a prior diagnosis of phenylketonuria (PKU) meeting the following criteria:
* Blood Phe concentration ≥600 µmol/L at screening;
* Average blood Phe concentration ≥600 μmol/L within the 12 months prior to screening (with at least two test results available);
* Genetic test report confirming the presence of phenylalanine hydroxylase (PAH) gene defects.
* Ability to maintain dietary stability from 3 days prior to screening through the end of the trial.
* Subjects (including partners) capable of practicing effective contraception during the trial period, with no plans for conception within 3 months after trial completion.
* Voluntarily sign the informed consent form and, in the investigator's judgment, able to comply with all study requirements.
Key Exclusion Criteria:
* Known hypersensitivity to PL54 injection or any of its excipients.
* Medical conditions or history assessed by the investigator that may increase the risk of severe allergic reactions (e.g., acute exacerbation of allergic asthma, atopic constitution, history of anaphylaxis, etc.).
* Use of any medication for PKU (including large neutral amino acids) within 14 days prior to study drug administration or within 5 half-lives of the drug (whichever is longer).
* Use or planned use of any injectable medication containing polyethylene glycol (PEG) (except the investigational drug) within 3 months prior to…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse Events (AEs)
Timeframe: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib