A Study of MZE782 in Adults With PKU

NCT07694440 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MZE782-201
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 2

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)

Keywords

PKU; Phenylketonuria

Outcome Measures

Primary Outcomes (2)

• Safety and tolerability based on incidence of adverse events (AEs)

  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

  Baseline and Weeks 2, 3, and 4

Secondary Outcomes (3)

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

  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

  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

  Baseline and Weeks 2, 3, 4, 8, 10, and 13

Study Arms (6)

MZE782 Low Dose (Cohort 1A)

EXPERIMENTAL

Drug: MZE782

Placebo (Cohort 1A)

PLACEBO COMPARATOR

Other: Placebo

MZE782 High Dose (Cohort 1B)

EXPERIMENTAL

Drug: MZE782

Placebo (Cohort 1B)

PLACEBO COMPARATOR

Other: Placebo

MZE782 High Dose (Cohort 2)

EXPERIMENTAL

Drug: MZE782

Placebo (Cohort 2)

PLACEBO COMPARATOR

Other: Placebo

Interventions

MZE782 DRUG

Tablet, Low Dose

MZE782 Low Dose (Cohort 1A)

Placebo OTHER

Tablet

Placebo (Cohort 1A); Placebo (Cohort 1B); Placebo (Cohort 2)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Maze Therapeutics: lead

Study Sites (2)

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

RECRUITING

Clinic for Special Children

Gordonville, Pennsylvania, 17529, United States

RECRUITING

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Maze Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Maze Therapeutics

CONTACT

650-850-5075; medicalinfo@mazetx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2026
• First Posted: July 10, 2026
• Study Start: June 23, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): February 1, 2028
• Last Updated: July 14, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)