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

NCT ID NCT07694440

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 10, 2026 · Last updated Aug 21, 2026 · Updated 4 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 38 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Feb 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Males and females 18 to 75 years old * Clinical diagnosis of PKU * Willing and able to keep diet and protein intake consistent during the study * For some study groups, is willing and able to stop certain PKU medications before starting study treatment * For some study groups, may need to meet additional blood phenylalanine level or background-treatment requirements * Meets minimum blood phenylalanine level requirements for study group Key Exclusion Criteria: * Is pregnant, breastfeeding, or planning to become pregnant or conceive a child during the study or within 90 days after the last dose * Medical conditions or abnormal test results that could make participation unsafe or make it difficult to complete study procedures or interpret study results * Has recently received another investigational drug * Is unable to swallow tablets or tolerate oral medication Other inclusion/exclusion criteria defined in protocol apply.

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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)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    6 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Clinic for Special Children

    RECRUITING

    Gordonville, Pennsylvania, 17529, United States

  • Indiana University School of Medicine

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Stanford University Clinical and Translational Research Unit

    RECRUITING

    Palo Alto, California, 94304, United States

  • UCSF Benioff Children's Hospital Oakland

    RECRUITING

    Oakland, California, 94609, United States

  • University of Kentucky Medical Center

    RECRUITING

    Lexington, Kentucky, 40536, United States

  • University of Tennessee Health Science Center Le Bonheur Children's Hospital

    RECRUITING

    Memphis, Tennessee, 38103, United States

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