Clinical Trials Logo

Clinical Trial Summary

This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children.

Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to < 2 years of age with either a diagnosed or clinically suspected UCD.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT02246218
Study type Interventional
Source Horizon Pharma Ireland, Ltd., Dublin Ireland
Contact
Status Completed
Phase Phase 4
Start date December 31, 2014
Completion date July 17, 2017

See also
  Status Clinical Trial Phase
Terminated NCT01948427 - Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Terminated NCT03933410 - UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder Phase 2
Completed NCT03064048 - Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD N/A
Recruiting NCT04612764 - Liver Disease in Urea Cycle Disorders
Completed NCT05330039 - Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
Completed NCT03721367 - Chronic Liver Disease in Urea Cycle Disorders
Completed NCT03911089 - A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD N/A
Terminated NCT03181828 - Manipulating the Gut Microbiome Study Phase 1/Phase 2
Completed NCT00472732 - Neurologic Injuries in Adults With Urea Cycle Disorders N/A
Recruiting NCT05076318 - Dysregulated Urea-synthesis at Terminal Uremia N/A
Completed NCT03179878 - Safety and Tolerability of SYNB1020-CP-001 Phase 1
Recruiting NCT04602325 - Systemic Biomarkers of Brain Injury From Hyperammonemia
Withdrawn NCT03884959 - A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients Phase 2
Recruiting NCT04908319 - Hepatic Histopathology in Urea Cycle Disorders
Completed NCT03335488 - Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) Phase 4
Completed NCT04248062 - Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism
Completed NCT05706714 - Th1, Th2, Th17 Phenotype in Urea Cycle Disorders
Terminated NCT03343756 - HepaStem Long-Term Safety Registry