Search Medical Condition
Please enter condition
Please choose location from dropdown

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.

RESULTS

Found (11) clinical trials

THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years. As an observational study, enrolled patients will not be required to make any additional office visits or take any medicine outside of normal care.

Phase N/A

UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care  

Phase

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

UCDs are a group of rare genetic diseases that affect how protein is broken down in the body. The cause of UCDs is a deficiency in one of eight enzymes responsible for removing ammonia, a waste product of protein metabolism, from the bloodstream. Normally, ammonia is converted into urea and ...

Phase N/A

Longitudinal Study of Urea Cycle Disorders

Urea cycle disorders are a group of rare genetic diseases that affect how protein is broken down in the body. UCDs are caused by a deficiency in one of six enzymes or two mitochondrial membrane transporters responsible for removing ammonia, a waste product of protein metabolism, from the bloodstream. Normally, ...

Phase N/A

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

Argininosuccinate lyase deficiency (ASLD; also known as argininosuccinic aciduria) is the second most common urea cycle disorder (UCD) and accounts for 15-20% of all disorders of ureagenesis. Individuals with ASLD can have unique clinical and physiologic characteristics as compared to other UCDs. Previous work from the members of the UCDC ...

Phase N/A

Nitric Oxide Supplementation in Argininosuccinic Aciduria

The investigators will perform a double blind, randomized, placebo-controlled, crossover study of NO supplementation in ASA patients assessing endothelial function and blood pressure as primary endpoints, and markers of nitric oxide production as exploratory endpoints. Subjects will receive a nitrite-restricted diet prior to the start and during the trial period. ...

Phase N/A

Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.

Phase

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

PROTOCOL OUTLINE: This protocol involves 2 clinical studies. Part A is a metabolic study of glutamine conversion to urea at different levels of protein intake, while on and off medications. Part B is a dose escalation study of a first-generation adenoviral vector with an E1 deletion and an E3 deletion ...

Phase

UNLOCKED: A Phase 2 Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder

We expect the trial to enroll approximately 24 adult Urea Cycle Disorder (UCD) patients on standard of care with elevated ammonia levels. The planned treatment duration is eight weeks, with a primary endpoint of plasma ammonia levels, as measured by plasma ammonia 24-hour area under the curve. Patients will also ...

Phase

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Na ve 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 subjects not currently or previously chronically treated with phenylacetic acid (phenylacetate; PAA) prodrugs. The study design will include: 1) Baseline Period; 2) ...

Phase