Urea Cycle Disorder Clinical Trials

A listing of Urea Cycle Disorder medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 13 clinical trials
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders

This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to NaPBA in urea cycle disorder

phenylbutyrate
vasectomy
deficiency
hysterectomy
genetic testing
  • 44 views
  • 03 May, 2022
  • 8 locations
Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

This is a study involving a dietary supplement. Patients with argininosuccinate lyase deficiency (ASLD) will be randomly assigned to receive either a nitric oxide dietary supplement or placebo for 24 weeks, and then crossed-over to receive the other treatment for 24 weeks. The investigators will assess the effects of the …

argininosuccinic aciduria
nitric oxide
deficiency
genetic testing
  • 26 views
  • 04 Oct, 2022
  • 1 location
Dysregulated Urea-synthesis at Terminal Uremia

This project will examine the dysregulation of the urea cycle in patients with terminal uremia using a validated method named "Functional Hepatic Nitrogen Clearance"

urea
chronic hemodialysis
Accepts healthy volunteers
  • 0 views
  • 22 Oct, 2021
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

  • 0 views
  • 19 Oct, 2022
  • 2 locations
Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.

  • 0 views
  • 07 Oct, 2022
  • 1 location
The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino Acids

The aim of this study is to reduce fasting circulating BCAA concentrations in volunteers using Sodium Phenylbutyrate. In addition, we aim investigate the effect of BCAA lowering on fasting blood glucose levels, insulin sensitivity measures and muscle metabolism.

  • 0 views
  • 08 Sep, 2021
  • 1 location
Longitudinal Study of Urea Cycle Disorders

Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy, and coma. The purpose of this

argininosuccinic aciduria
deficiency
citrullinemia type ii
ornithine
citrulline
  • 100 views
  • 23 Jan, 2022
  • 16 locations
Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease

The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease

  • 37 views
  • 16 Oct, 2022
  • 4 locations
Liver Disease in Urea Cycle Disorders

This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE

  • 0 views
  • 15 Mar, 2022
  • 2 locations
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders (UCD)

In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) which perturbation is thought to be

argininosuccinic aciduria
encephalopathy
deficiency
cognitive impairment
arginosuccinate
  • 75 views
  • 16 Feb, 2022
  • 1 location