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Tests treatment safety and results for Phenylketonuria (PKU)
Official title A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
ClinicalTrials.gov ID: NCT07672756
What this study is testing
What is PL54?
PL54 is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for phenylketonuria (pku).
Also referred to as PEGylated Recombinant Phenylalanine Ammonia-Lyase.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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?
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 55
You may be able to join if
- Adult patients aged 18 to 55 years, inclusive, regardless of sex.
- people 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.
You likely can't join if
- 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...
- 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...
- Use or planned use of any injectable medication containing polyethylene glycol (PEG) (except the investigational drug) within 3 months prior to...
- 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...
See the full eligibility criteria
- Adult patients aged 18 to 55 years, inclusive, regardless of sex.
- people 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.
- people (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
- 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...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Hefei, Anhui, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Hefei, Anhui, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.