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Completed

NCT Number: NCT00654004

Fatty Acid Oxidation Disorders & Body Weight Regulation Grant

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.

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Key information

Conditions

Trifunctional Protein Deficiency Body Weight Pathological Conditions, Signs and Symptoms Signs and Symptoms Trifunctional Protein Deficiency With Myopathy And Neuropathy

Age range

7 year–40 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Oregon Health & Science University

Portland, Oregon, 97239, United States

About this study

A role for mitochondrial fatty acid oxidation in the peripheral signaling cascade of leptin, adiponectin and insulin has recently been proposed from animal studies but has not been investigated in humans. Children with trifunctional protein (TFP, including deficiency of long-chain hydroxyacyl-CoA dehydrogenase) and very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, inherited disorders of long-chain fatty acid ß-oxidation, lack an ability to oxidize fatty acids for energy. They have increased levels of body fat and circulating leptin and a high incidence of obesity. Current therapy for children with these disorders is based on frequent meals and consuming a low fat, very high carbohydrate diet. Despite treatment, exercise induced rhabdomyolysis is a common complication of TFP and VLCAD deficiency that frequently leads to exercise avoidance. The effects of these genetic defects on body composition and weight regulation have not been investigated. The contribution of fatty-acid oxidation during moderate intensity exercise in children has also not been reported.

Two groups of subjects were recruited: one group of subjects had a long-chain fatty acid oxidation disorder (n=13). The other group is a group of controls (n=16). We studied peripheral signals of body weight regulation, glucose tolerance, body composition, and exercise metabolism in subjects with a long-chain fatty acid oxidation disorder compared to normal controls.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • confirmed diagnosis of TFP, LCHAD, CPT2 or VLCAD deficiency
  • at least 7 years of age
  • willingness to complete overnight admission
  • generally healthy

Exclusion criteria

  • inclusion in another research project that alters macronutrient intake
  • diabetes, thyroid disease or other endocrine dysfunction that alters body composition.
  • pregnancy
  • anemia

Treatment and study plan

Primary outcomes

  1. An Outcome of This Study is the Difference in Percent Body Fat (%BF) Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Normal Controls.

    Time frame: Subjects will be compared to controls at one point in time.

    Body composition by DEXA was measured in subjects with a long-chain fatty acid oxidation disorder (n=13). Twelve age, sex and BMI matched controls and 4 heterozygotes for a long-chain fatty acid oxidation disorder were recruited who also completed body composition measures. The difference in body composition between subjects and age matched controls was compared by t-test.

  2. An Outcome of This Study is the Difference in Glucose Tolerance Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Normal Controls.

    Time frame: Subjects will be compared to controls at one point in time.

    Glucose tolerance was estimated by the Matsuda Index using glucose and insulin values from a standard oral glucose tolerance test. The Matsuda Index is calculated by the following formula: 10,000/ sq root of (fasting glucose mg/dl X fasting insulin in units/ml) X (mean glucose (mg/dl) X mean insulin (units/ml) and correlates with insulin sensitivity measured by the gold standard method of a hyperinsulinemic euglycemic clamp. Values of 2.5 or greater are considered insulin sensitive. Values of 2.4 or less are considered insulin resistance.

    The Matsuda Index of Insulin Sensitivity was measured in subjects with a long-chain fatty acid oxidation disorder (n=12). Twelve age, sex and BMI matched controls and 4 heterozygotes for a long-chain fatty acid oxidation disorder were recruited who also completed an oral glucose tolerance test. The difference in Mastuda Index between subjects and age matched controls was compared by t-test.

Secondary outcomes

  1. The Difference in Plasma Adiponectin Levels Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Matched Controls Was Compared by T-test

    Time frame: Fasting total adiponectin (ug/ml)

    Fasting total adiponectin levels in ug/ml were measured in both groups (subjects with a long-chain fatty acid oxidation disorder). The differences between groups were compared with a t-test

  2. The Difference in Plasma Leptin Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Matched Controls Was Compared by T-test

    Time frame: Fasting leptin levels ng per kg of fat mass

    Fasting leptin in ng/kg fat mass were measured in both groups (subjects with a long-chain fatty acid oxidation disorder; controls). The differences between groups were compared with a t-test

  3. The Difference in Plasma Insulin Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Matched Controls Was Compared by T-test

    Time frame: Fasting insulin levels uUnits/ml

    Fasting insulin levels in uU/ml were measured in both groups. The differences between groups were compared with a t-test

Sponsors and collaborators

Lead sponsor

Oregon Health and Science University

Other

Collaborators

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Oregon State University

Registry information

Official study title

Fatty Acid Oxidation Disorders & Body Weight Regulation

Important dates

Study start
2006
Primary completion
2010
Study completion
2011
First posted
Apr 7, 2008
Registry last updated
Apr 22, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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