New PKU pill enters early human testing
NCT ID NCT07241234
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
RECRUITINGSzczecin, West Pomeranian Voivodeship, 71-252, Poland
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Indiana University
RECRUITINGIndianapolis, Indiana, 46202, United States
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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University of Texas Southwestern Medical Center (UTSW)
RECRUITINGDallas, Texas, 75390, United States