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Protein Deficiency clinical trials

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NCT ID: NCT02517307 Completed - Normal Volunteers Clinical Trials

Fatty Acid Oxidation Defects and Insulin Sensitivity

Start date: February 2016
Phase: N/A
Study type: Interventional

The purpose of this study is to learn more about what causes insulin resistance. It has been suggested that proper breakdown of fat into energy (oxidation) in the body is important to allow insulin to keep blood sugar in the normal range. The investigators want to know if having one of the fatty acid oxidation disorders could have an influence on insulin action. Fatty acid oxidation disorders are genetic disorders that inhibit one of the enzymes that converts fat into energy. The investigators will study both normal healthy people and people with a long-chain fatty acid oxidation disorder.

NCT ID: NCT01668186 Recruiting - Clinical trials for RCDP - Rhizomelic Chondrodysplasia Punctata

Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Start date: January 2012
Phase:
Study type: Observational

The Peroxisome Biogenesis Disorders (PBD) are a group of inherited disorders due to defects in peroxisome assembly causing complex developmental and metabolic sequelae. In spite of advancements in peroxisome biology, the pathophysiology remains unknown, the spectrum of phenotypes poorly characterized and the natural history not yet systematically reported. Our aims are to further define this population clinically, biochemically and genetically. The investigators will prospectively follow patients from Canada, the US and internationally, and collect data from medical evaluations, blood, urine and imaging studies that would be performed on a clinical care basis. For patients who are unable to attend our clinic, we will collect all medical records and images since birth as well as subsequent records/images for the next 5 years or until the end of the study. Clinical data from medical records will be banked in our Peroxisomal Disorder Research Databank and Biobank. The investigators will use this information to identify standards of care and improve management.

NCT ID: NCT01494051 Completed - Clinical trials for Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency

High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders

Start date: January 2006
Phase: Phase 1/Phase 2
Study type: Interventional

The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.

NCT ID: NCT01461304 No longer available - Barth Syndrome Clinical Trials

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Start date: n/a
Phase:
Study type: Expanded Access

This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.

NCT ID: NCT01399333 Recruiting - Protein Deficiency Clinical Trials

Comparison of Meal Replacements Versus Protein Supplements and Utilizing the Protein Digestibility Corrected Amino Acid Score (PDCAAS) Method

PDCAAS
Start date: January 2010
Phase: Phase 4
Study type: Interventional

The purpose of this research study is to compare the varying qualities of protein supplements utilizing the Protein Digestibility Corrected Amino Acid Score (PDCAAS) method and evaluating the clinical outcome related to protein stores and absorption.

NCT ID: NCT00840112 Terminated - Clinical trials for Peripheral Neuropathy

Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy

Start date: July 2010
Phase: N/A
Study type: Interventional

Purpose: People with a genetic defect in the ability to burn fat can also develop a problem with the nerves in their feet. The nerve problem, or neuropathy, can limit their ability to walk. Part of the treatment of their genetic defect in the ability to burn fat is to eat a very low fat diet. Vitamin E is found only in fatty foods like oils and nuts. People with a genetic defect in the ability to burn fat may have low vitamin E because of their low fat diet. The purpose of this study is to test whether vitamin E supplements can improve the nerve function in the feet of people with a genetic defect in the ability to burn fat. Procedures: Blood samples will be drawn at the beginning of the study, after 2 months and after 6 months of vitamin E supplements. The blood will be analyzed for plasma vitamin E concentrations. Around the time of each blood draw subjects will record all the food and beverages he or she consumes for three days. The subject will send the record to the investigator. Subjects will have a physical exam by a doctor specializing in nerves, a neurologist before and after taking vitamin E. They will have nerve function measured with a test called a nerve conduction velocity or NCV. Subjects will be given 800 international units (IU) of vitamin E per day for 6 months.

NCT ID: NCT00654004 Completed - Clinical trials for Trifunctional Protein Deficiency

Fatty Acid Oxidation Disorders & Body Weight Regulation Grant

Start date: April 2006
Phase: N/A
Study type: Observational

Several hormones involved in body weight regulation increase the subject's ability to burn fat for energy. The purpose of this study is to investigate how burning fat for energy may affect those hormones and body weight in children. The study will also determine if eating a diet higher in protein alters the amount of fat you burn and how these hormones control body weight.