Phase 1 Phenylketonuria Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You are between 18 and 55 years old (inclusive), of any sex
• You have previously been diagnosed with phenylketonuria (PKU), a condition affecting how your body processes a substance called phenylalanine
• Your blood phenylalanine (Phe) level was 600 µmol/L or higher at your screening appointment
• Your average blood Phe level over the past 12 months was also 600 µmol/L or higher, with at least two test results on record
• You have a genetic test report confirming you carry a fault in the gene responsible for PKU (the PAH gene)
• You are able to keep your diet consistent starting 3 days before screening through to the end of the trial
• You and/or your partner are willing and able to use effective birth control throughout the trial and for at least 3 months after it ends, with no plans to try to conceive during that time
• You are willing to sign a consent form and, in the doctor's opinion, able to follow all the study requirements

Who may not be able to join:

• You have a known allergy to the study drug (PL54 injection) or any of its ingredients
• You have a medical history that the doctor believes could increase your risk of a serious allergic reaction, such as severe allergic asthma, a general tendency toward allergies, or a previous history of severe allergic reactions (anaphylaxis)
• You have taken any PKU medication (including large neutral amino acids) in the 14 days before receiving the study drug, or within a certain washout period based on the medication used (whichever is longer)
• You have used, or plan to use, any injectable medication containing a substance called polyethylene glycol (PEG) — such as a certain type of contraceptive injection — within 3 months before screening or during the study (not including the study drug itself)
• You have previously been treated with a drug called Pegvaliase (brand name PALYNZIQ®)
• You have previously had a whole-body reaction to a product containing PEG, such as breathing problems, stomach/gut symptoms, low blood pressure, swelling, or an allergic reaction — the doctor will assess this based on how serious your previous symptoms were
• You have abnormal results from vital signs checks, physical examinations, or lab tests at screening that the doctor believes make it unsafe for you to participate
• You have a significant medical history involving your immune system, heart, lungs, digestive system, hormones, kidneys, blood, nervous system, mental health, or a history of cancer (except certain types of skin or cervical cancer that have been fully removed with no signs of return)
• You have been regularly taking immune-suppressing medications (such as cyclosporine or azathioprine) within 4 weeks before screening and are unable to stop taking them during the study
• You have received a vaccine within 4 weeks before screening, or have a vaccination planned during the study period
• You have tested positive for syphilis, HIV, hepatitis C, or hepatitis B
• Your kidney function test (creatinine) result is more than 1.5 times the upper limit of the normal range
• Your liver function test (ALT) result is more than 2 times the upper limit of the normal range
• You are currently pregnant or breastfeeding
• You have taken part in another clinical trial and received an experimental treatment within the 3 months before screening
• You have previously received any gene therapy for PKU, including as part of a clinical study
• The doctor has any other reason to believe it would not be safe or appropriate for you to join the trial

Important: Always verify eligibility with the trial site directly before applying.