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New oral drug aims to tame PKU's toxic protein buildup

NCT ID NCT07694440

Recruiting now Disease control Sponsor: Maze Therapeutics Source: ClinicalTrials.gov ↗

First seen Jul 10, 2026 · Last updated Jul 15, 2026 · Updated 2 times

Summary

This study tests an experimental oral drug called MZE782 in adults with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down an amino acid called phenylalanine (Phe). High Phe levels can cause brain damage. The trial compares two doses of MZE782 against a placebo to see if the drug safely lowers blood Phe levels. Participants must maintain a consistent diet and may need to pause other PKU medications during the study.

What this could mean

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

Active substance
MZE782
What this could lead to
If successful, MZE782 could offer a new oral treatment option to help control phenylalanine levels in adults with PKU, potentially easing dietary restrictions.
What could go wrong
This is an early phase 2 trial with only 38 participants, so results may not apply to everyone. The drug may cause side effects or fail to lower phenylalanine enough to be clinically meaningful.

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 (PKU)

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Clinic for Special Children

    RECRUITING

    Gordonville, Pennsylvania, 17529, United States

  • Indiana University School of Medicine

    RECRUITING

    Indianapolis, Indiana, 46202, United States

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