Recruiting
Phase 2

MZE782

Sponsor:

Maze Therapeutics

Code:

NCT07694440

Conditions

Phenylketonuria (PKU)

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

MZE782

Placebo

MZE782

Study Details

Brief summary:

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

Conditions

Phenylketonuria (PKU)

Study ID

NCT07694440

Start date

Jun 23, 2026

Status verified date

Jul, 2026

Completion date

Feb, 2028

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

38 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: MZE782 Low Dose (Cohort 1A)

placebo comparator: Placebo (Cohort 1A)

experimental: MZE782 High Dose (Cohort 1B)

placebo comparator: Placebo (Cohort 1B)

experimental: MZE782 High Dose (Cohort 2)

placebo comparator: Placebo (Cohort 2)

Interventions

MZE782

Tablet, Low Dose

Placebo

Tablet

MZE782

Tablet, High Dose

Primary outcome measure

  • Safety and tolerability based on incidence of adverse events (AEs) [ Time Frame: Week 0 to Week 17 ]
  • For Cohorts 1A and 1B: Absolute change in plasma Phe levels from Baseline to the mean of plasma Phe levels [ Time Frame: Baseline and Weeks 2, 3, and 4 ]

Central Contacts and Locations

Central contacts

Maze Therapeutics

650-850-5075medicalinfo@mazetx.com

Locations

UCSF Benioff Children's Hospital Oakland

Recruiting

Oakland, California, United States, 94609

Stanford University Clinical and Translational Research Unit

Recruiting

Palo Alto, California, United States, 94304

Indiana University School of Medicine

Recruiting

Indianapolis, Indiana, United States, 46202

University of Kentucky Medical Center

Recruiting

Lexington, Kentucky, United States, 40536

Clinic for Special Children

Recruiting

Gordonville, Pennsylvania, United States, 17529

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

Recruiting

Memphis, Tennessee, United States, 38103

More Information

Sponsor

Maze Therapeutics

Last update posted

Aug 20, 2026

Last verified

Jul, 2026

Keywords

  • PKU
  • Phenylketonuria

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-11. This information was provided to ClinicalTrials.gov by Maze Therapeutics on 2026-08-20.