Who may be eligible for the A Study of MZE782 in Adults With PKU trial?

Key Inclusion Criteria: * Males and females 18 to 75 years old * Confirmed 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 * 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.

ClinicalTrials.gov record NCT07694440

A Study of MZE782 in Adults With PKU

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Oct 10, 2026

A Phase 2, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety, Tolerability, and Efficacy of MZE782 in Adults With Phenylketonuria (PKU)

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.

Age18 years old to 75 years old

SexAll

LocationUnited States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2 study tests the drug MZE782 in adults with phenylketonuria (PKU). Participants are randomly assigned to low- or high-dose MZE782 or matching placebo in parallel groups, with quadruple masking. The study measures safety and tolerability and tracks plasma phenylalanine and plasma MZE782 concentrations during the treatment period (primary safety timeframe to Week 17).

What it is studying. The study is testing whether MZE782 tablets are safe and tolerable in adults with PKU and how the drug affects blood (plasma) phenylalanine levels and plasma concentrations of MZE782.

What the researchers hope to learn. Scientific goals are to evaluate the safety and tolerability of MZE782, to assess changes in plasma phenylalanine levels from baseline, and to measure plasma concentrations (pharmacokinetics) of MZE782 across dose groups; the study also analyzes proportions of participants reaching specified plasma Phe thresholds.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: MZE782
Other: Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Males and females 18 to 75 years old
Confirmed 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
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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

If you take part you will be randomly assigned to take oral tablets of MZE782 (at different doses) or a matching placebo and will take study medication during the trial. The study involves repeated blood tests to measure plasma phenylalanine (Phe) and MZE782 drug concentrations at baseline and at several scheduled visits (for example Weeks 1, 2, 3, 4, 8, 10, and 13), and safety will be monitored through Week 17 with recording of adverse events. Participants must keep diet and protein intake consistent during the study; some groups must stop certain PKU medications before starting; the study excludes people who are pregnant or breastfeeding and requires being able to swallow oral tablets.

How the study is structured. This is a randomized, parallel-group, placebo-controlled Phase 2 trial with dose-ranging cohorts. Participants are assigned to low-dose or high-dose MZE782 or to matching placebo; the study is quadruple-masked so participants, care providers, investigators, and outcome assessors do not know who receives drug or placebo. The trial measures adverse events for safety, plasma phenylalanine levels at specified time points, and plasma concentrations of MZE782 across weeks noted in the outcomes (primary safety timeframe covers Week 0 to Week 17).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on phenylketonuria

Clinical trials38 trials listed for phenylketonuriaEvery trial the registry lists for phenylketonuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in phenylketonuria researchLatest monthly update: September 2026. Includes: Diet supply gaps. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Jun 23, 2026
Primary completion: December 2027 (estimated)
Study completion: February 2028 (estimated)
Target enrollment: 38 participants (estimated)
Last updated on registry: Oct 8, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Maze Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Maze Therapeutics 📞 650-850-5075 ✉ medicalinfo@mazetx.com

Participating sites (7)

UCSF Benioff Children's Hospital OaklandRecruiting
Oakland, California, 94609 · United States
Stanford University Clinical and Translational Research UnitRecruiting
Palo Alto, California, 94304 · United States
Ann and Robert H. Lurie Children's Hospital of ChicagoRecruiting
Chicago, Illinois, 60611 · United States
Indiana University School of MedicineRecruiting
Indianapolis, Indiana, 46202 · United States
University of Kentucky Medical CenterRecruiting
Lexington, Kentucky, 40536 · United States
Clinic for Special ChildrenRecruiting
Gordonville, Pennsylvania, 17529 · United States
University of Tennessee Health Science Center Le Bonheur Children's HospitalRecruiting
Memphis, Tennessee, 38103 · United States

Terms used on this page · explanations by MedicalUpdates.ai

study group: One of the groups in a study. Each arm receives a particular intervention, comparator or no intervention, so the groups can be compared.
investigational drug: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
exclusion criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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