Phase 1b Study to Assess Safety, Tolerability, and Effects of AG-181 in Adults with Phenylketonuria

What this Trial Is About

Researchers are evaluating the safety and tolerability of AG-181 in adults with Phenylketonuria PKU, a genetic condition affecting the metabolism of phenylalanine. This Phase 1b study aims to understand how the drug behaves in the body, including how it is processed and its effects on phenylalanine levels. The study is open-label and involves participants with specific genetic mutations related to PKU and elevated phenylalanine levels. Participants will receive AG-181 tablets daily from Day 1 to Day 28. The study includes up to two cohorts, both receiving the investigational drug, with dosing and treatment occurring over the 28-day period. The study is non-randomized and open-label, focusing on evaluating safety, drug levels in the blood, and pharmacodynamics during this treatment phase. During the study, participants will undergo assessments to monitor adverse events and serious side effects up to Day 42. Blood samples will be taken up to Day 28 to measure drug concentration, peak levels, time to peak concentration, overall exposure, clearance, volume of distribution, and changes in phenylalanine levels. These evaluations help researchers understand the drugs safety profile and its impact on the metabolic condition over the treatment duration.

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria