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 have been diagnosed with PKU and genetic testing shows you carry two specific gene changes in the PAH gene, with at least one being a change called R408W (this will be confirmed by the trial's own lab during the screening process).
• At least once in the past year, a blood test showed your phenylalanine (Phe) levels were above 600 μmol/L (a measure of a substance that builds up in PKU).
• During the trial's screening period, your average blood Phe level was above 600 μmol/L, and no single reading was below 360 μmol/L.
• Your body weight is at least 50 kg (about 110 lbs), and your body mass index (BMI — a measure based on height and weight) is between 18.0 and 35.0 during the screening period.
• A dietitian has confirmed in writing that you are able to keep your diet consistent in protein and phenylalanine throughout the study, as the trial requires.

Who may not be able to join:

• You have previously received a treatment called AG-181.
• You are currently taking certain medications that affect how drugs move through the body (called P-gp inhibitors) and have not stopped them at least 5 days (or long enough based on that medicine) before starting the trial drug.
• You are currently taking certain other medications that strongly affect how the body processes drugs (called strong CYP1A2, CYP2C8, or CYP3A inhibitors or inducers) and have not stopped them at least 28 days before starting the trial drug.
• You are currently taking medicines that reduce stomach acid, such as proton pump inhibitors or H2 blockers (note: some short-acting antacids like calcium carbonate may be allowed — confirm with trial site).
• You have a pre-existing condition that the doctor believes could affect how your digestive system absorbs or processes the trial drug (confirm with trial site).
• You have a pre-existing condition that the doctor believes could affect how your liver or kidneys process the trial drug (confirm with trial site).
• You are unable to swallow or take medication by mouth.
• You are unwilling to stop taking existing PKU treatments — such as sapropterin (Kuvan), pegvaliase (Palynziq), or any other PKU therapy — by a specific point during the screening period (around 30 days before the trial starts).

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