Browse Studies — Arctuva

Phase 3

A Phase 1/2/3 First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzy…

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseOrnithine Carbamoyltransferase Deficiency (Disorder)+1 more

Estimated

~$10 – $8,750

iECURE, Inc.NCT06255782

Phase 2

A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency

Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.

OTC DeficiencyOrnithine Transcarbamylase DeficiencyOTCD

Estimated

~$10 – $8,750

Arcturus Therapeutics, Inc.NCT06488313

Hepatic Histopathology in Urea Cycle Disorders

This is a multi-site, retrospective chart review as well as a prospective study to evaluate histopathologic findings in liver samples from individuals with any…

Urea Cycle DisorderOrnithine Transcarbamylase DeficiencyCitrullinemia 1+7 more

Estimated

~$10 – $750

Baylor College of MedicineNCT04908319