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Trial record 1 of 6 for:    children | Long chain 3 hydroxyacyl-CoA dehydrogenase deficiency OR Trifunctional protein deficiency (TFP) | Child
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Fatty Acid Oxidation Disorders & Body Weight Regulation Grant

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ClinicalTrials.gov Identifier: NCT00654004
Recruitment Status : Completed
First Posted : April 7, 2008
Results First Posted : April 22, 2013
Last Update Posted : April 22, 2013
Sponsor:
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Oregon State University
Information provided by (Responsible Party):
Melanie B Gillingham, Oregon Health and Science University

Tracking Information
First Submitted Date April 3, 2008
First Posted Date April 7, 2008
Results First Submitted Date December 11, 2012
Results First Posted Date April 22, 2013
Last Update Posted Date April 22, 2013
Study Start Date April 2006
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 18, 2013)
  • 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.
  • 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.
Original Primary Outcome Measures
 (submitted: April 3, 2008)
The primary 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: December 2009 ]
Change History Complete list of historical versions of study NCT00654004 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: April 18, 2013)
  • 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
  • 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
  • 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
Original Secondary Outcome Measures
 (submitted: April 3, 2008)
The secondary outcome of this study is the difference in weight gain between subjects with TFP and VLCAD deficiency consuming a high protein compared to a high carbohydrate diet for four months. [ Time Frame: December 2009 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Fatty Acid Oxidation Disorders & Body Weight Regulation Grant
Official Title Fatty Acid Oxidation Disorders & Body Weight Regulation
Brief Summary 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.
Detailed Description

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.

Study Type Observational
Study Design Observational Model: Case Control
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples Without DNA
Description:
Blood and urine samples. Body composition and energy expenditure data. MRI/MRS images.
Sampling Method Non-Probability Sample
Study Population Subjects were patients with a diagnosis of mitochondrial trifunctional protein, long-chain 3-hydroxyacylCoA dehydrogenase, very long-chain acylCoA dehydrogenase or carnitine palmitoyltransferase 2 deficency. They were recruited through advertisements on the FAO support website, or physician referral. Control subjects were from the greater Portland area. They were recruited via adverstisements at OHSU.
Condition Trifunctional Protein Deficiency
Intervention Not Provided
Study Groups/Cohorts
  • Subjects
    Subjects are patients with a long-chain fatty acid oxidation disorder including CPT2, VLCAD, TFP or LCHAD deficiency.
  • Controls
    Subjects do not have a fatty acid oxidation disorder.
Publications * Behrend AM, Harding CO, Shoemaker JD, Matern D, Sahn DJ, Elliot DL, Gillingham MB. Substrate oxidation and cardiac performance during exercise in disorders of long chain fatty acid oxidation. Mol Genet Metab. 2012 Jan;105(1):110-5. doi: 10.1016/j.ymgme.2011.09.030. Epub 2011 Oct 1.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: April 18, 2013)
26
Original Estimated Enrollment
 (submitted: April 3, 2008)
48
Actual Study Completion Date January 2011
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 7 Years to 40 Years   (Child, Adult)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00654004
Other Study ID Numbers DK71869
K01DK071869 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Melanie B Gillingham, Oregon Health and Science University
Study Sponsor Oregon Health and Science University
Collaborators
  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Oregon State University
Investigators
Principal Investigator: Melanie B. Gillingham, PhD Oregon Health and Science University
PRS Account Oregon Health and Science University
Verification Date April 2013