Advertisement

Topics

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

2014-08-27 03:58:19 | BioPortfolio

Summary

RATIONALE: The urea cycle is the process in which nitrogen is removed from the blood and converted into urea, a waste product found in urine . Urea cycle disorders are inherited disorders caused by the lack of an enzyme that removes ammonia from the bloodstream. Gene therapy is treatment given to change a gene so that it functions normally. Studying the treatment and metabolism of patients with urea cycle disorders may be helpful in developing new treatments for these disorders.

PURPOSE: Two-part clinical trial to study the treatment and metabolism of patients who have urea cycle disorders.

Description

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 substitution (d1309) expressing ornithine transcarbamylase (OTC).

In Part A, diet is controlled for protein and calories. Intravenous glutamine and urea are administered. Controls are given intravenous arginine, phenylacetate, and benzoate.

In Part B, groups of 3 patients are given a single low, intermediate, or high dose of intravenous OTC vector. Allopurinol is administered every 12 hours for 12 days. As of 12/10/1999, Part B of the study is closed.

Study Design

Primary Purpose: Treatment

Conditions

Amino Acid Metabolism, Inborn Errors

Intervention

Protein and calorie controlled diet, Ornithine transcarbamylase vector

Location

Baylor College of Medicine
Houston
Texas
United States
77030

Status

Recruiting

Source

National Center for Research Resources (NCRR)

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:58:19-0400

Clinical Trials [1941 Associated Clinical Trials listed on BioPortfolio]

Effects of Hypoxia and Inflammation on Citrulline Synthesis by Ornithine Transcarbamylase in Human Enterocytes

This study aims at exploring the effects of hypoxia and inflammation on the production of citrulline by ornithine transcarbamylase (OTC) activity in enterocytes from explant cultures of du...

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Rate-limiting Amino Acids in Endurance-trained Athlete

Protein requirements in individuals who participate in endurance-based exercise training have been suggested to be greater than the current recommended dietary allowance (RDA). The biolog...

A Controlled Trial of Protein Enrichment of Meal Replacements for Weight Reduction With Retention of Lean Body Mass

Study hypothesis: High protein diets have been shown to make people full and and help to retent of muscle mass. This study was designed to determine effects of a protein- enriched meal rep...

Safety and Efficacy of High Protein Diet Versus GFR Based Protein Diet in Heart Failure Patients With Renal Insufficiency

Effect of high calorie high protein diet versus high calorie,glomerular filtration rate (GFR) based protein intake in non-obese advanced HF patients will be assessed. In this regard, prote...

PubMed Articles [19397 Associated PubMed Articles listed on BioPortfolio]

Comparative folding analyses of unknotted versus trefoil-knotted ornithine transcarbamylases suggest stabilizing effects of protein knots.

Ornithine transcarbamylases (OTCs) are conserved enzymes involved in arginine biosynthesis in microbes and the urea cycle in mammals. Recent bioinformatics analyses identified two unique OTC variants,...

Effects of 2-day calorie restriction on cardiovascular autonomic response, mood, and cognitive and motor functions in obese young adult women.

Although long-term energy restriction has been widely investigated and has consistently induced improvements in health and cognitive and motor functions, the responses to short-duration calorie restri...

The Efficacy of Energy-Restricted Diets in Achieving Preoperative Weight Loss for Bariatric Patients: a Systematic Review.

In bariatric practice, a preoperative weight loss of at least 5% is recommended. However, the hypocaloric diets prescribed vary and no consensus exists. This study examined the efficacy of preoperativ...

Improving Weight Loss by Combination of Two Temporary Antiobesity Treatments.

It is generally accepted that the efficacy with an intragastric balloon decreases after the first 4 months of treatment. Aim of this study is to evaluate if the association of a very low-calorie keto...

Rational engineering of ornithine decarboxylase with greater selectivity for ornithine over lysine through protein network analysis.

Ornithine decarboxylase (ODC) converts C5 ornithine into C4 putrescine, a monomer for polyamide synthesis. However, ODC also has minor activity towards cell metabolite C6 lysine and yields C5 cadaveri...

Medical and Biotech [MESH] Definitions

A pyridoxal phosphate enzyme that catalyzes the formation of glutamate gamma-semialdehyde and an L-amino acid from L-ornithine and a 2-keto-acid. EC 2.6.1.13.

A high-affinity, low capacity system y+ amino acid transporter with strong similarity to CATIONIC AMINO ACID TRANSPORTER 1. The two isoforms of the protein, CAT-2A and CAT-2B, exist due to alternative mRNA splicing. The transporter has specificity for the transport of ARGININE; LYSINE; and ORNITHINE.

An inherited disorder due to defective reabsorption of CYSTINE and other BASIC AMINO ACIDS by the PROXIMAL RENAL TUBULES. This form of aminoaciduria is characterized by the abnormally high urinary levels of cystine; LYSINE; ARGININE; and ORNITHINE. Mutations involve the amino acid transport protein gene SLC3A1.

A diet that contains limited amounts of protein. It is prescribed in some cases to slow the progression of renal failure. (From Segen, Dictionary of Modern Medicine, 1992)

A pyridoxal-phosphate protein, believed to be the rate-limiting compound in the biosynthesis of polyamines. It catalyzes the decarboxylation of ornithine to form putrescine, which is then linked to a propylamine moiety of decarboxylated S-adenosylmethionine to form spermidine. EC 4.1.1.17.

More From BioPortfolio on "Study of Treatment and Metabolism in Patients With Urea Cycle Disorders"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topics

Urology
Benign Prostatic Hyperplasia (BPH) Erectile Dysfunction Urology Urology is the branch of medicine concerned with the urinary tract and diseases that affect it. Examples include urethritis, urethrostenosis and incontinence. Urology is a su...

Enzymes
Enzymes are proteins that catalyze (i.e., increase the rates of) chemical reactions. In enzymatic reactions, the molecules at the beginning of the process, called substrates, are converted into different molecules, called products. Almost all chemical re...


Searches Linking to this Trial