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AG-181 for Phenylketonuria (PKU) Study

This study is testing a new drug called AG-181 in people with Phenylketonuria (PKU). PKU is a genetic condition where the body can't break down a substance called phenylalanine. The main goal of this study is to see if AG-181 is safe and how well people tolerate it. Researchers will be looking at any side effects that happen. You can join if you are between 18 and 69 years old and have a confirmed diagnosis of PKU, specifically with at least one R408W mutation in your PAH gene. The study plans to enroll about 20 participants. The current recruitment status is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 20 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Researchers will track side effects for up to 42 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07241234

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

Recruiting
PHASE1Ages 18–69InterventionalTreatment
Agios Pharmaceuticals, Inc.
~20 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:AG-181

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Subjects with Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to the Study Drug
Measured over Up to Day 42

Conditions

Phenylketonuria

Explore related trials

PhenylketonuriaPAH Gene

Where it's being run

4 sites across 4 states
Indiana1
Pennsylvania1
Texas1
West Pomeranian Voivodeship1

Who to contact

Agios Medical Affairs
[email protected]
833-228-8474
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Diagnosis of PKU, defined as documented presence of 2 mutant alleles in the phenylalanine hydroxylase (PAH) gene, of which at least 1 is the R408W mutation, as determined during Screening per the genotyping performed by the study central genotyping laboratory.
At least 1 plasma Phe concentration greater than (\>) 600 micromoles per liter (μmol/L) in the 52 weeks before providing informed consent.
Average concentration of plasma Phe \> 600 μmol/L in Phe samples taken during Screening, with no individual assessment below 360 μmol/L. Any Phe samples taken after Day -20 will not be included.
Body mass index (BMI) greater than or equal to (≥) 18.0 kilograms per meter square (kg/m\^2) to lesser than or equal to (≤) 35.0 kg/m\^2 and weight ≥ 50 kilograms (kg) at any time during the Screening Period.
Documented approval from a dietitian confirming that the subject can maintain their diet consistent in protein and Phe intake throughout the study as outlined in the Diet Manual.

Exclusion

Prior exposure to AG-181.
Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥ 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug.
Receiving products that are strong inhibitors or strong inducers of cytochrome P450 CYP1A2, CYP2C8, or CYP3A that have not been stopped for ≥ 28 days before administration of the first dose of study drug.
Receiving treatment with an acid-reducing agent, including but not limited to proton pump inhibitors and H2 blockers. Short-acting acid-reducing agents such as calcium carbonate are permitted.
Any preexisting condition that could (in the opinion of the Investigator) interfere with gastrointestinal anatomy or motility that may disrupt the absorption, metabolism, and/or excretion of the study drug.
Any preexisting condition that could (in the opinion of the Investigator) interfere with hepatic or renal function that may disrupt the absorption, metabolism, and/or excretion of the study drug.
Inability to tolerate oral medication.
Unwillingness to washout from tetrahydrobiopterin (BH4) supplementation (eg, sapropterin dihydrochloride, Kuvan), pegvaliase-pqpz (Palynziq), or any other PKU therapy by Day -30 during Screening.

What this trial measures

  • Number of Subjects with Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to the Study DrugUp to Day 42