Urea Cycle Disorders, Inborn: all 59 studies

Page 1 of 1. Open to participants first (13), then studies with results posted, then the rest; newest registration first within each.

• MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)Not yet recruitingNCT07892651
• Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCDRecruitingNCT07773246Phase 1/2
• Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving TaskRecruitingNCT07701694
• Study of LNP.UCD.ABE in Patients With Urea Cycle DisordersRecruitingNCT07667387Phase 1/2
• At Home Ammonia Monitoring of Inborn Errors of Ammonia MetabolismRecruitingNCT06953505Not applicable
• A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle DisordersRecruitingNCT06904027
• Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)RecruitingNCT06805695
• MyRareDiet A Novel Diet Tracking ToolNot yet recruitingNCT06664840Not applicable
• Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder PatientsRecruitingNCT05671666Not applicable
• Hepatic Histopathology in Urea Cycle DisordersRecruitingNCT04908319
• Systemic Biomarkers of Brain Injury From HyperammonemiaRecruitingNCT04602325
• Neuroimaging and Neuropsychological Outcomes in Urea Cycle DisordersRecruitingNCT02935283
• Longitudinal Study of Urea Cycle DisordersRecruitingNCT00237315
• Chronic Liver Disease in Urea Cycle DisordersCompletedNCT03721367Results posted
• Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)CompletedNCT03335488Phase 4Results posted
• Manipulating the Gut Microbiome StudyTerminatedNCT03181828Phase 1/2Results posted
• PCORI Urea Cycle Disorder StudyCompletedNCT02740153Results posted
• A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle DisordersCompletedNCT02246218Phase 4Results posted
• Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle DisordersCompletedNCT02111200Not applicableResults posted
• Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)TerminatedNCT01948427Results posted
• Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)CompletedNCT01347073Phase 3Results posted
• To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)CompletedNCT01257737Phase 4Results posted
• Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle DisordersCompletedNCT00992459Phase 3Results posted
• Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle DisordersCompletedNCT00947544Phase 2Results posted
• Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)CompletedNCT00947297Phase 3Results posted
• Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle DisordersCompletedNCT00551200Phase 2Results posted
• Neurologic Injuries in Adults With Urea Cycle DisordersCompletedNCT00472732Results posted
• Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle DisorderCompletedNCT00345605Phase 2Results posted
• An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC DeficiencyActive, not recruitingNCT06255782Phase 3
• Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC GenotypeActive, not recruitingNCT06247670Phase 1
• Th1, Th2, Th17 Phenotype in Urea Cycle DisordersCompletedNCT05706714
• Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)CompletedNCT05330039
• Dysregulated Urea-synthesis at Terminal UremiaCompletedNCT05076318Not applicable
• Liver Disease in Urea Cycle DisordersActive, not recruitingNCT04612764
• Patient and Observer Reported Outcome Measurements in Inborn Errors of MetabolismCompletedNCT04248062
• UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle DisorderTerminatedNCT03933410Phase 2
• A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCDCompletedNCT03911089Not applicable
• A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric PatientsWithdrawnNCT03884959Phase 2
• Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle DisordersCompletedNCT03797131Not applicable
• Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC DeficiencyActive, not recruitingNCT03636438
• HepaStem Long-Term Safety RegistryTerminatedNCT03343756
• Safety and Tolerability of SYNB1020-CP-001CompletedNCT03179878Phase 1
• Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLDCompletedNCT03064048Not applicable
• Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)CompletedNCT02489292Phase 2
• Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes AssessmentCompletedNCT02311283
• Nitric Oxide Supplementation in Argininosuccinic AciduriaCompletedNCT02252770Not applicable
• Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo ModelsCompletedNCT02124330Not applicable
• Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)CompletedNCT02051049
• Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)CompletedNCT01765283Phase 1/2
• Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young InfantsCompletedNCT01624311Phase 2
• Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle FunctionCompletedNCT01549015Not applicable
• Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle DisordersTerminatedNCT01541722
• The NIH UNI Study: Urea Cycle Disorders, Nutrition and ImmunityTerminatedNCT01421888
• Increasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateWithdrawnNCT01341379Phase 2
• Human Heterologous Liver Cells for Infusion in Children With Urea Cycle DisordersTerminatedNCT01195753Phase 2
• Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity AssessmentCompletedNCT01002469Not applicable
• A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control GroupCompletedNCT00986895Phase 1
• Human Heterologous Liver Cells for Infusion in Children With Urea Cycle DisordersCompletedNCT00718627Phase 2
• Study of Treatment and Metabolism in Patients With Urea Cycle DisordersStatus unknownNCT00004307Phase 1

← Urea Cycle Disorders, Inborn overview