Phase 2a Study to Assess Safety and Effects of ARCT-810 in Adolescents and Adults With OTC Deficiency

What this Trial Is About

Researchers are evaluating the safety and pharmacodynamics of multiple doses of ARCT-810, an investigational drug, in adolescents and adults with Ornithine Transcarbamylase OTC deficiency. This Phase 2a, open-label study aims to better understand how ARCT-810 works and its safety profile in people aged 12 years and older who have this condition. Participants must have a documented diagnosis of OTC deficiency and be medically managed with a stable diet and supplements. Participants will receive up to five intravenous infusions of ARCT-810 every two weeks, following a diet stabilization period of at least four weeks. The study includes three different dose levels, and dose escalation or cohort expansion may occur after three participants complete each dose level. Participants will continue their usual clinical management for OTC deficiency throughout the study. During the study, participants will have clinic visits at screening and on Days 1, 15, 29, 36, 43, 57, 60, 71, and 85. Researchers will monitor adverse events, pharmacokinetics, fasting plasma ammonia, plasma glutamine, and stable isotope ureagenesis assay values. The primary outcome is the incidence, severity, and dose relationship of adverse events by Day 85. The total participation duration includes ongoing assessments up to Day 85 to evaluate safety and drug effects.

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD