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The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MZE782 Low Dose (Cohort 1A) | Experimental |
| |
| Placebo (Cohort 1A) | Placebo Comparator |
| |
| MZE782 High Dose (Cohort 1B) | Experimental |
| |
| Placebo (Cohort 1B) | Placebo Comparator |
| |
| MZE782 High Dose (Cohort 2) | Experimental |
| |
| Placebo (Cohort 2) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MZE782 | Drug | Tablet, Low Dose |
| |
| Placebo |
| Measure | Description | 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 |
| Measure | Description | 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 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other inclusion/exclusion criteria defined in protocol apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Maze Therapeutics | Contact | 650-850-5075 | medicalinfo@mazetx.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Maze Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinic for Special Children | Recruiting | Gordonville | Pennsylvania | 17529 | United States |
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| ID | Term |
|---|---|
| D010661 | Phenylketonurias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Other |
Tablet |
|
| MZE782 | Drug | Tablet, High Dose |
|
| 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 |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |