A Study of MZE782 in Adults With PKU

Phase 2RecruitingNCT07694440

Maze Therapeutics38 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Phenylketonuria (PKU)

Interventions

MZE782DRUG

Tablet, Low Dose

PlaceboOTHER

Tablet

MZE782DRUG

Tablet, High Dose

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Locations (6)

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

RECRUITING

Stanford University Clinical and Translational Research Unit

Palo Alto, California, United States

RECRUITING

Indiana University School of Medicine

Indianapolis, Indiana, United States

Outcomes

Primary Outcomes

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

Secondary Outcomes

For Cohorts 1A and 1B, analyzed separately: Geometric mean plasma concentrations of MZE782

Time frame: Weeks 1, 2, 3, 4, 8, and 13

For Cohorts 1A and 1B, analyzed separately: Absolute and Percent change in plasma Phe levels from Baseline to the mean of plasma Phe levels

Time frame: Baseline up to Week 13

For Cohorts 1A and 1B, analyzed separately: Proportion of participants with mean plasma Phe levels ≤600 μMol/L, ≤360 μMol/L, and ≤120 μMol/L

Time frame: Baseline and Weeks 2, 3, 4, 8, 10, and 13

Central Contacts

Maze Therapeutics

CONTACT

650-850-5075 medicalinfo@mazetx.com

Data from ClinicalTrials.gov

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