A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Trial Parameters
Brief Summary
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Eligibility Criteria
Inclusion Criteria: 1. Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent. 2. Males and Females aged ≥12 years, at Screening. 3. Documented clinical diagnosis of OTC deficiency. 4. History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening. 5. Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days. 6. Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range). 7. Must be willing to adhere to contraception guidelines. Exclusion Criteria: 1. Uncontrolled hypertension. 2. Symptoms of infection for at least 7 days prior to dosing. 3. Malignancy within 5 years, except for basal or squamous cell carcinoma