Phase 2 Trial of MZE782 Tablets for Safety and Effectiveness in Adults Aged 18 to 75 with Phenylketonuria PKU

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of MZE782 tablets in adults aged 18 to 75 years who have Phenylketonuria PKU, a genetic condition affecting amino acid metabolism. This Phase 2 study aims to assess how well MZE782 works and how safe it is for people with PKU by comparing different doses of the drug to a placebo. The study is sponsored by Maze Therapeutics and uses a randomized, parallel design with multiple groups. Participants will receive either a low or high dose of MZE782 or a matching placebo tablet. The treatment period lasts up to 17 weeks, during which plasma phenylalanine Phe levels and drug concentrations will be regularly measured. Some study groups may have specific requirements regarding blood Phe levels or background treatments, including stopping certain PKU medications before starting the study drug. The study includes multiple cohorts receiving different doses or placebo, with evaluations at baseline and multiple weeks throughout treatment. During the trial, participants will have regular blood tests to measure Phe levels and monitor drug levels at several time points up to week 13. Researchers will track safety by recording any adverse events from the start through week 17. Participants must maintain a consistent diet and protein intake during the study. The total participation lasts several months, with close monitoring to assess both the impact of MZE782 on PKU and its safety profile.

A Study of MZE782 in Adults With PKU