Phenylketonuria (PKU)
Conditions
Keywords
PKU, Phenylketonuria
Brief summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
Interventions
DRUGMZE782
Tablet, Low Dose
OTHERPlacebo
Tablet
Sponsors
Maze Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Contacts
CONTACTMaze Therapeutics
STUDY_DIRECTORMedical Director
Maze Therapeutics
Outcome results
None listed