Trials / Conditions / Ornithine Transcarbamylase Deficiency

Ornithine Transcarbamylase Deficiency

19 registered clinical trials studyying Ornithine Transcarbamylase Deficiency — 7 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP) NCT06805695	iECURE, Inc.	—
Recruiting	A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD NCT06488313	Arcturus Therapeutics, Inc.	Phase 2
Recruiting	An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC D NCT06255782	iECURE, Inc.	Phase 1 / Phase 2
Recruiting	Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn NCT05092685	University College, London	Phase 1 / Phase 2
Terminated	Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerabilit NCT05526066	Arcturus Therapeutics, Inc.	Phase 2
Unknown	Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan NCT05910151	West Kazakhstan Medical University	—
Completed	Baby Detect : Genomic Newborn Screening NCT05687474	Centre Hospitalier Universitaire de Liege	—
Recruiting	Hepatic Histopathology in Urea Cycle Disorders NCT04908319	Baylor College of Medicine	—
Recruiting	Liver Disease in Urea Cycle Disorders NCT04612764	Baylor College of Medicine	—
Terminated	Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease NCT04909346	Ultragenyx Pharmaceutical Inc	—
Completed	Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With NCT04442347	Arcturus Therapeutics, Inc.	Phase 1
Terminated	Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency NCT04717453	Ultragenyx Pharmaceutical Inc	—
Completed	Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects NCT04416126	Arcturus Therapeutics, Inc.	Phase 1
Withdrawn	Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Defici NCT03767270	Translate Bio, Inc.	Phase 1 / Phase 2
Completed	A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult NCT04269122	Ultragenyx Pharmaceutical Inc	—
Enrolling By Invitation	Early Check: Expanded Screening in Newborns NCT03655223	RTI International	—
Completed	Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, an NCT01569568	Andrea Gropman	—
Completed	Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders NCT00718627	Cytonet GmbH & Co. KG	Phase 2
Completed	Neurologic Injuries in Adults With Urea Cycle Disorders NCT00472732	Andrea Gropman	—