Clinical Trials Logo
  1. Home
  2. Other
  3. NCT04717453

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

Ornithine Transcarbamylase Deficiency Clinical Trial

Official title:
A Study to Characterize Rate of Ureagenesis Utilizing Oral [1-13C] Sodium Acetate in the Spectrum of Severity of Patients With Ornithine Transcarbamylase (OTC) Deficiency

Clinical Trial Summary

The objectives of the study are to characterize urea production rates in patients with OTC, characterize the association of rate of ureagenesis and disease severity in OTC patients, characterize the association of rate of ureagenesis and executive and verbal function and characterize the association of rate of ureagenesis and patient-reported functional status.

Clinical Trial Description

Study DTX301-CL102 is a noninterventional, observational study to characterize the rate of ureagenesis and to assess neurocognition and functional status in the spectrum of OTC deficiency and their association with biochemical characteristics. [1-13C]Sodium acetate will be administered orally as a tracer to measure the rate of ureagenesis. ;

Study Design

Related Conditions & MeSH terms

  • Ornithine Transcarbamylase Deficiency
Clinical Trial Details
NCT number NCT04717453
Study type Observational
Source Ultragenyx Pharmaceutical Inc
Contact
Status Terminated
Phase
Start date October 6, 2020
Completion date December 15, 2021
View Details
See also
  Status Clinical Trial Phase
Completed NCT01569568 - Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT05910151 - Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT04612764 - Liver Disease in Urea Cycle Disorders
Active, not recruiting NCT04442347 - Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency Phase 1
Not yet recruiting NCT02670889 - Urease Inhibitor Drug Treatment for Urea Cycle Disorders Phase 1/Phase 2
Recruiting NCT05092685 - Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn Phase 1/Phase 2
Completed NCT00472732 - Neurologic Injuries in Adults With Urea Cycle Disorders N/A
Terminated NCT04909346 - Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Completed NCT04416126 - Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects Phase 1
Recruiting NCT05526066 - Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 Phase 2
Completed NCT00718627 - Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Phase 2
Recruiting NCT04908319 - Hepatic Histopathology in Urea Cycle Disorders
Withdrawn NCT03767270 - Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency Phase 1/Phase 2
Completed NCT04269122 - A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
Recruiting NCT06255782 - An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE) Phase 1/Phase 2