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This is a Phase 1, randomized, double-blind, placebo-controlled, single (SAD) / multiple (MAD) ascending dose and food effect study.
Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered MZE001 in healthy subjects, in fasted and fed states. Each SAD and MAD cohort will enroll 8 subjects randomized 6 active: 2 placebo to receive single or multiple doses of MZE001.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MZE001 | Experimental | MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease. |
|
| Placebo | Placebo Comparator | Excipients containing no active ingredients. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MZE001 | Drug | Small molecule inhibitor of muscle glycogen synthase |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events as a measure of safety and tolerability of MZE001 | Occurrence of adverse events, serious adverse events, adverse events of special interest | 14 days |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum concentration following multiple doses of MZE001 | PK | 14 days |
| AUC following multiple doses of MZE001 | PK | 14 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sarah Noonberg, MD | Maze Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Orange County Research Center | Tustin | California | 92780 | United States |
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| ID | Term |
|---|---|
| D008353 | Mannitol |
| C031183 | stearic acid |
| ID | Term |
|---|---|
| D013402 | Sugar Alcohols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D002241 | Carbohydrates |
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| Placebo | Drug | Product containing excipients with no active ingredients |
|
|
| Accumulation ratio following multiple doses of MZE001 | PK | 14 days |