Ornithine Transcarbamylase Deficiency Clinical Trials

25 clinical trials for Ornithine Transcarbamylase Deficiency across international registries, including 6 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency — RECRUITING · PHASE3
• Hepatic Histopathology in Urea Cycle Disorders — RECRUITING
• A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD — RECRUITING · PHASE2
• Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP) — RECRUITING
• Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn — RECRUITING · PHASE1
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ADENOASSOCIATED VIRUS SEROTYPE 8 (AAV8)-MEDIATED GENE TRANSFER OF HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) IN PATIENTS WITH LATE-ONSET OTC DEFICIENCY — RECRUITING · PHASE3
• 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 — TERMINATED · PHASE2
• Early Check: Expanded Screening in Newborns — ACTIVE_NOT_RECRUITING
• Long-term Follow-up (LTFU) Study of Participants in any iECURE Protocol Using an Investigational Product — TERMINATED · PHASE2
• Liver Disease in Urea Cycle Disorders — ACTIVE_NOT_RECRUITING
• Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 — TERMINATED · PHASE2
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency — COMPLETED · PHASE1
• Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI — COMPLETED
• Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects — COMPLETED · PHASE1
• Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan — OTHER
• Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease — TERMINATED
• Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency — TERMINATED
• A PHASE 1B RANDOMIZED, DOUBLE BLINDED, PLACEBO CONTROLLED, ASCENDING DOSE STUDY TO ASSESS THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF SINGLE DOSES OF ARCT-810 IN CLINICALLY STABLE PATIENTS WITH ORNITHINE TRANSCARBAMYLASE DEFICIENCY — NOT_YET_RECRUITING · PHASE1
• A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects — COMPLETED
• Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency — WITHDRAWN · PHASE1
• A LONG-TERM FOLLOW-UP STUDY TO EVALUATE THE SAFETY AND EFFICACY OF ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 (AAV8)-MEDIATED GENE TRANSFER OF HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) IN ADULTS WITH LATE-ONSET OTC DEFICIENCY — COMPLETED
• A PHASE 1/2, OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 (AAV8)-MEDIATED GENE TRANSFER OF HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) IN ADULTS WITH LATE-ONSET OTC DEFICIENCY — COMPLETED · PHASE1
• Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders — COMPLETED · PHASE2
• Neurologic Injuries in Adults With Urea Cycle Disorders — COMPLETED

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