Phase 2 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 willing to take part and have signed the consent form agreeing to join the study
• You have been diagnosed with PKU caused by having two faulty copies of the PAH gene (confirmed by genetic testing)
• You are an adult between 18 and 55 years old
• Your blood phenylalanine (Phe) levels have been measured at 600 μmol/L (10 mg/dL) or higher on at least two occasions in the past 24 months, with at least one of those measurements taken within the last 6 months
• You are willing and able to follow a managed diet as part of the study
• You are willing and able to follow all study procedures and requirements, in the opinion of the study doctor
• If you are a woman who could become pregnant, you must have a negative pregnancy blood test within 7 days before receiving the treatment, and you must agree to use highly effective contraception for at least one year after receiving the study treatment

Who may not be able to join:

• You have antibodies in your blood that could interfere with the study's gene therapy delivery system (known as anti-AAV8 neutralizing antibodies at a level of 1:5 or higher)
• Your PKU is already well managed with current treatments, such as Sapropterin (Kuvan) or Pegvaliase (Palynziq)
• Your blood test results before the treatment show that your liver, kidney, or blood cell levels are outside acceptable ranges — including abnormal liver enzymes, bilirubin, kidney function, blood clotting, blood sugar, or blood counts (confirm specific values with trial site)
• You have significant abnormal results from health checks, tests, or physical exams that the study doctor feels make you unsuitable
• In the study doctor's opinion, you have a condition or reason that means you should not take corticosteroids (steroid medicines), or steroids could make your condition worse
• You currently have or have had Hepatitis A, B, or C, HIV, syphilis, or active or latent tuberculosis
• You have had a significant liver condition — such as fatty liver disease, liver scarring, or liver inflammation — within the 6 months before signing the consent form (except for a condition called Gilbert's syndrome)
• You have a history of cancer (malignant tumours)
• A liver scan (ultrasound) shows signs of serious liver disease such as significant scarring or cirrhosis
• You have a history of serious heart, lung, digestive, hormone, kidney, blood, nerve, mental health, or other major health conditions, in the opinion of the study doctor
• You have a known allergy to human serum albumin
• You have a history of substance misuse, such as alcohol or drug abuse
• You have previously received any form of gene therapy
• You have taken part in another clinical drug trial and received that trial's drug within the past 3 months (or within 5 half-lives of that drug, whichever is longer)
• You have elevated levels of a protein called Alpha-fetoprotein (AFP) in your blood (confirm with trial site)
• You have serious health complications related to PKU, such as kidney problems, osteoporosis, severe anaemia, major depression, or epilepsy, or other conditions the study doctor feels make you unsuitable
• You weigh more than 100 kg
• Your daily diet includes more than 2 g of natural protein per kg of body weight per day

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