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 and able to sign a consent form agreeing to take part, or someone legally authorised can sign on your behalf
• You have been diagnosed with "classic PKU," a specific form of phenylketonuria caused by certain gene mutations that mean your body produces little or no PAH enzyme
• You are an adult between 18 and 55 years old at the time of signing the consent form
• You have tried available PKU treatments (such as Kuvan or Palynziq) but they have not worked for you, or you cannot tolerate them
• If you were previously on PKU medications but stopped (for medical reasons or your own choice), you must have been off them for at least 28 days before signing the consent form
• You have had at least one recorded blood phenylalanine (Phe) level of 600 μmol/L or higher while eating your normal diet in the past 6 months
• You are willing to keep a food diary and follow guidance from a dietitian during the trial
• You are able and willing to follow all study procedures in the opinion of the study doctor
• If you are a woman who could become pregnant, you must have a negative pregnancy blood test before joining, and agree to use a reliable form of contraception for at least 1 year after receiving the study treatment (acceptable methods include vasectomy, condoms, hormonal contraception, an IUD, or true and consistent abstinence — periodic abstinence such as calendar methods is not accepted)
• If you are a male participant, you must use contraception until at least 3 consecutive semen samples show no trace of the study treatment's viral component

Who may not be able to join:

• Your PKU is caused by something other than a PAH gene mutation
• Your blood tests show antibodies that could block the study treatment (anti-AAV8 neutralising antibodies)
• Two consecutive blood tests before the study show that your liver function or blood count results are outside the acceptable ranges set by the trial (confirm specific values with trial site)
• Your vital signs — such as temperature, blood pressure, heart rate, breathing rate, or oxygen levels — are outside normal ranges at screening, and the study doctor considers this a concern
• You have a medical reason that would make taking steroid medication (corticosteroids, used as part of this trial) unsafe or unsuitable for you
• You have an active infection including hepatitis A, B, or C, HIV, tuberculosis, or syphilis
• You have a history of significant liver disease such as fatty liver disease, scarring of the liver (fibrosis), or liver cirrhosis in the past 6 months (Gilbert's syndrome is an exception — confirm with trial site)
• You have or have had any form of cancer, past or present
• A liver ultrasound has shown liver scarring, cirrhosis, or other serious liver disease
• You have a serious condition affecting your heart, lungs, kidneys, digestive system, hormones, blood, nerves, or mental health
• You have a known allergy to human albumin (a protein used in some medical products)
• You have a substance use disorder (for example, dependence on alcohol, heroin, or amphetamines)
• You have previously received any gene therapy at any point in your life
• You have taken part in another clinical trial or received an experimental treatment within the past 3 months (or longer, depending on the treatment)
• Your blood test shows a raised level of a protein called alpha-fetoprotein (AFP)
• You have other serious PKU-related health conditions — such as kidney problems, osteoporosis, anaemia, severe acid reflux or stomach ulcers, major depression, or epilepsy — that the study doctor believes could be made worse by the trial treatment
• You are currently taking any PKU medication, such as Kuvan or Palynziq
• You weigh more than 120 kg
• You eat more than 2 g of natural protein per kilogram of your body weight each day
• You are currently breastfeeding

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