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Arginase Deficiency Clinical Trials

A listing of Arginase Deficiency 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 (10) clinical trials

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments. Subjects will be randomized to ...

Phase

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency

This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single IV doses of DTX301. Eligible subjects will receive a single IV infusion of DTX301. Dose ...

Phase

Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism

Background Limitations to the body of evidence on effects of interventions on important outcome parameters in IEM and thus to evidence-based recommendations or guidelines are manifold. Due to the rarity of each disease, knowledge about the natural history may be poor. Most IEM are clinically heterogeneous with severity of the ...

Phase N/A

Arginase Inhibitor INCB001158 as a Single Agent and in Combination With Immune Checkpoint Therapy in Patients With Advanced/Metastatic Solid Tumors

This study is an open-label Phase 1 evaluation of INCB001158 as a single agent and in combination with immune checkpoint therapy in patients with advanced/metastatic solid tumors. Single Agent INCB001158: Patients with advanced/metastatic solid tumors will be enrolled into escalating monotherapy dose cohorts to determine the Recommended Phase 2 Dose ...

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

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

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

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

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

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