New PKU pill enters early human testing

NCT ID NCT07241234

Recruiting now Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 31, 2026 · Updated 3 times

Summary

This early-stage trial is testing a new drug called AG-181 in 20 adults with phenylketonuria (PKU), a genetic condition that makes it hard to break down an amino acid called phenylalanine. The main goal is to check if the drug is safe and tolerable. Researchers will also measure how the drug moves through the body. Participants must have a specific genetic mutation (R408W) and high phenylalanine levels.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: AG-181 (a drug taken as a tablet)
What this could lead to: If successful, this could lead to a new treatment option to help control phenylalanine levels in people with PKU.
What could go wrong: This is an early Phase 1 trial with only 20 participants, so it is primarily testing safety. It may not show enough benefit or could have side effects. Much more research is needed.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIA

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
RECRUITING

Szczecin, West Pomeranian Voivodeship, 71-252, Poland
Indiana University
RECRUITING

Indianapolis, Indiana, 46202, United States
UPMC Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern Medical Center (UTSW)
RECRUITING

Dallas, Texas, 75390, United States

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