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Phase 1
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
iECURE, Inc.NCT06255782
Longitudinal Study of Urea Cycle Disorders
Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy…
Brain Diseases, Metabolic, InbornAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders
Andrea GropmanNCT00237315
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) w…
Urea Cycle Disorders
Children's National Research InstituteNCT02935283