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NCT Number: NCT07694440

A Study of MZE782 in Adults With PKU

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

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Key information

Conditions

Phenylketonuria (PKU) Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Phenylketonurias

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Indiana University School of Medicine, Indianapolis, Indiana, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

MZE782

Drug

Tablet, Low Dose

Placebo

Other

Tablet

Primary outcomes

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

    Time frame: Week 0 to Week 17

  2. 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

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

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

  2. 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

  3. 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

Study contacts

Contact information is provided by the study sponsor or research team.

Maze Therapeutics

CONTACT

[email protected]

650-850-5075

Sponsors and collaborators

Lead sponsor

Maze Therapeutics

Industry

Registry information

Official study title

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)

Acronym: CIPheR

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Jul 10, 2026
Registry last updated
Jul 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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