Phenylketonuria (PKU)Phase 1NCT07672756
A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
A Phase 1 study testing PL54 in people with Phenylketonuria (PKU), run by Chongqing Peg-Bio Biopharm Co., Ltd. at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Phenylketonuria (PKU)
- What is being tested?
- PL54
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 20 Jul 2025to 1 Aug 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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 screening or during the study period, including medroxyprogesterone acetate injection.
- Prior use of Pegvaliase (PALYNZIQ®).
- History of any of the following manifestations associated with prior PEG-containing product use: systemic symptoms (e.g., respiratory or gastrointestinal symptoms, hypotension, angioedema, allergic reactions). The investigator will determine eligibility based on the severity of previous symptoms.
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- Clinically significant abnormal vital signs, physical examination, laboratory tests, or other relevant findings at screening, deemed by the investigator to render the subject unsuitable for enrollment.
- Significant medical history of immunological, cardiovascular, respiratory, gastrointestinal, endocrine, renal, hematological, neurological, psychiatric, or malignant diseases (except basal cell carcinoma of the skin or carcinoma in situ of the cervix that has been excised with no evidence of recurrence) assessed by the investigator prior to screening.
- Stable use of immunosuppressants (e.g., cyclosporine, azathioprine) within 4 weeks prior to screening, with inability to discontinue use during the study period.
- Vaccination within 4 weeks prior to screening or planned vaccination during the study period.
- Positive serology for Treponema pallidum-specific antibodies, human immunodeficiency virus (HIV) antibodies, hepatitis C virus (HCV) antibodies, or hepatitis B virus (HBV) surface antigen.
- Serum creatinine ratio > 1.5 × upper limit of normal (ULN).
- Alanine aminotransferase (ALT) > 2 × ULN.
- Pregnant or breastfeeding women.
- Participation in any investigational drug trial and receipt of an investigational product within 3 months prior to screening.
- Prior use of any gene therapy for phenylketonuria (including participation in clinical studies).
- Any other condition deemed by the investigator to preclude participation in the trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230022
Huan Zhou, 86-13983367811, sunj@pegbiocq.com
Jianping Weng
About this study
Official title: A Phase I Multicenter Open-label Dose-escalation Study of Safety, Tolerability, PK/PD and Immunogenicity of Single/Multiple Subcutaneous PL54 Injection in Adult PKU Patients
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?
Read more
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.
Read the detailed description
This is a multicenter, open-label, dose-escalation Phase I clinical study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of single or multiple subcutaneous injections of PL54 injection in adult patients with phenylketonuria (PKU). The trial is divided into Phase Ia and Phase Ib.
Phase Ia:
A single subcutaneous injection will be administered. The phase includes a 2-week screening period, a single administration on Day 1 (D1), and a 4-week follow-up period, totaling approximately 6 weeks. Approximately 30 subjects will be enrolled across five dose groups: 0.01 mg/kg, 0.04 mg/kg, 0.1 mg/kg, 0.25 mg/kg, and 0.5 mg/kg, with 6 subjects per group (including 1 sentinel). The first subject in each dose group will receive a single subcutaneous injection of PL54, and the remaining subjects in the same dose group will be enrolled only after the first subject has completed at least 14 days of safety assessment. Dose escalation to the next group will proceed only after all subjects in the previous dose group have completed at least 14 days of visit assessments and the Safety Monitoring Committee (SMC) determines that the dose escalation termination criteria have not been met.
Phase Ib:
Approximately 18 subjects will be enrolled. Subcutaneous injections will be administered once weekly for 4 consecutive weeks (the dosing period for subsequent dose groups may be adjusted based on observed anti-drug antibody [ADA], pharmacokinetic [PK], and pharmacodynamic [PD] results from previous dose groups). Three dose groups are planned: 0.04 mg/kg, 0.1 mg/kg, and 0.25 mg/kg, with 6 subjects per group (the actual dose groups and dosing regimen will be adjusted based on Phase Ia results). Dose escalation to the next group will proceed only after all subjects in the previous dose group have completed at least 28 days of visit assessments and the SMC determines that the dose escalation termination criteria have not been met.
- Conditions
- Phenylketonuria (PKU)
- Being tested
- PL54
- Keywords
- Phenylketonuria, PKU
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PL54Biological
Also called PEGylated Recombinant Phenylalanine Ammonia-Lyase
Phase Ia:
Single, subcutaneous injection administration. Dose escalation will start from the initial dose of 0.01 mg/kg and proceed sequentially to higher doses.
Phase Ib:
Administration via subcutaneous injection. Dosing frequency: once weekly. Dose escalation will start from the initial dose of 0.04 mg/kg and proceed sequentially to higher doses.
| Study group | Role | What they receive |
|---|---|---|
| PL54 | Experimental | subcutaneous injection administration. Single -dose escalation will start from the initial dose of 0.01 mg/kg and proceed sequentially to higher doses. Receives: Biological: PL54 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs)
including the incidence of overall and categorized AEs
Measured: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
Secondary outcomes (4)
The change in blood phenylalanine concentration from baseline
Comparing the average phenylalanine concentrations at different timepoints and different doses with the concentration at baseline in Phase Ia and Phase Ib studies.
Measured: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
The proportion of subjects whose blood phenylalanine concentration decreased to the target range (60-600 μmol/L)
Monitor the blood phenylalanine concentration at each visit and calculate the proportion of subjects whose phenylalanine concentration drops to the target range (60-600 μ mol/L) for each dose group in Phase Ia and Phase Ib studies.
Measured: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
PK parameters of PL54 after single dose and multiple doses
Calculate the following PK parameters, including but not limited to Cmax, Tmax, AUC0-t, AUC0-∞, T1/2, λz, CL/F, Vz/F for Phase Ia, and AUC0-τ, Cmin,ss, Cmax,ss, Tmax,ss, T1/2 for Phase Ib.
Measured: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
Immunogenicity of PL54
Total anti-drug antibodies (if applicable), anti-PAL4 antibodies, anti-PEG IgG/IgM, neutralizing antibodies that inhibit PL54 activity (NAbs), and detection of anti-PL54 IgE antibodies in subjects experiencing severe allergic reactions (WAO criteria).
Measured: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2026
- Study start20 Jul 2025
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Phenylketonuria (PKU)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chongqing Peg-Bio Biopharm Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07672756
- Sponsor's protocol ID
- PL54-A101
- Status last verified
- Jun 2026
- First submitted
- 17 Jun 2026
- Last updated
- 1 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Multicenter Open-label Dose-escalation Study of Safety, Tolerability, PK/PD and Immunogenicity of Single/Multiple Subcutaneous PL54 Injection in Adult PKU Patients
- ClinicalTrials.gov ID
- NCT07672756
- Acronym / sponsor ID
- PL54-A101
- Conditions
- Phenylketonuria (PKU)
- Keywords
- Phenylketonuria, PKU
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Chongqing Peg-Bio Biopharm Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Jul 2025 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Aug 2027 (estimated)
- First posted
- 29 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a multicenter, open-label, dose-escalation Phase I clinical study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of single or multiple subcutaneous injections of PL54 injection in adult patients with phenylketonuria (PKU). The trial is divided into Phase Ia and Phase Ib.
Phase Ia:
A single subcutaneous injection will be administered. The phase includes a 2-week screening period, a single administration on Day 1 (D1), and a 4-week follow-up period, totaling approximately 6 weeks. Approximately 30 subjects will be enrolled across five dose groups: 0.01 mg/kg, 0.04 mg/kg, 0.1 mg/kg, 0.25 mg/kg, and 0.5 mg/kg, with 6 subjects per group (including 1 sentinel). The first subject in each dose group will receive a single subcutaneous injection of PL54, and the remaining subjects in the same dose group will be enrolled only after the first subject has completed at least 14 days of safety assessment. Dose escalation to the next group will proceed only after all subjects in the previous dose group have completed at least 14 days of visit assessments and the Safety Monitoring Committee (SMC) determines that the dose escalation termination criteria have not been met.
Phase Ib:
Approximately 18 subjects will be enrolled. Subcutaneous injections will be administered once weekly for 4 consecutive weeks (the dosing period for subsequent dose groups may be adjusted based on observed anti-drug antibody [ADA], pharmacokinetic [PK], and pharmacodynamic [PD] results from previous dose groups). Three dose groups are planned: 0.04 mg/kg, 0.1 mg/kg, and 0.25 mg/kg, with 6 subjects per group (the actual dose groups and dosing regimen will be adjusted based on Phase Ia results). Dose escalation to the next group will proceed only after all subjects in the previous dose group have completed at least 28 days of visit assessments and the SMC determines that the dose escalation termination criteria have not been met.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PL54 | Experimental | Biological: PL54subcutaneous injection administration. Single -dose escalation will start from the initial dose of 0.01 mg/kg and proceed sequentially to higher doses. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Adult patients aged 18 to 55 years, inclusive, regardless of sex.
- Subjects with a prior diagnosis of phenylketonuria (PKU) meeting the following criteria:
- 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 (17 listed)
- 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 screening or during the study period, including medroxyprogesterone acetate injection.
- Prior use of Pegvaliase (PALYNZIQ®).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PL54Also: PEGylated Recombinant Phenylalanine Ammonia-LyasePhase Ia: Single, subcutaneous injection administration. Dose escalation will start from the initial dose of 0.01 mg/kg and proceed sequentially to higher doses. Phase Ib: Administration via subcutaneous injection. Dosing frequency: once weekly. Dose escalation will start from the initial dose of 0.04 mg/kg and proceed sequentially to higher doses. | Biological | PL54 |
Primary outcome
Adverse Events (AEs)
Time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
including the incidence of overall and categorized AEs
4 secondary outcomes
The change in blood phenylalanine concentration from baseline
Time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
The proportion of subjects whose blood phenylalanine concentration decreased to the target range (60-600 μmol/L)
Time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
PK parameters of PL54 after single dose and multiple doses
Time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
Immunogenicity of PL54
Time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PL54 in people with Phenylketonuria (PKU), run by Chongqing Peg-Bio Biopharm Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jing Sun (86-13983367811, sunj@pegbiocq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (AEs) (time frame: Day 1-29 of Phase Ia; Day 1-57 of Phase Ib). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2026.