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

Study Overview
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Brief Summary
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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.

Detailed Description
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At baseline, the amount of muscle and fat in the whole body and inside the liver and muscle will be measured. All subjects will complete a moderate treadmill exercise test and a test to determine how they use sugar (oral glucose tolerance test). The amount and type of energy burned by each subject will be measured. Subjects will be randomly assigned to follow either a high carbohydrate diet or a reduced carbohydrate, high protein diet for 4 months. At the end of 4 months, the tests will be repeated.

Official Title
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Fatty Acid Oxidation and Body Weight Regulation in Long-chain Fatty Acid Oxidation Disorders.

Conditions
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Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency

Intervention / Treatment
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* Behavioral: Diet counseling


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: diagnosis of a long-chain fatty acid oxidation disorder including VLCAD, CPT2, LCHAD or TFP deficiency 7 years old or greater able to comply with diet guidelines Exclusion Criteria: pregnant enrolled in another study that alters diet composition cannot complete treadmill exercise study

Ages Eligible for Study
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Minimum Age: 7 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: High carbohydrate diet<br>Diet composition of 10% long-chain fatty acids, 20% medium-chain triglycerides, 12% protein and 68% carbohydrate is the current standard of care in long-chain fatty acid oxidation disorders. | Behavioral: Diet counseling<br>* Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home.<br>* Other names: high protein diet;|
| Experimental: High protein diet<br>Diet composition of 10% long-chain fatty acids, 20% medium chain triglycerides, 25% protein and 45% carbohydrate is the comparison diet. Fat content is the same between treatments; only the carbohydrate to protein ratio varies. | Behavioral: Diet counseling<br>* Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home.<br>* Other names: high protein diet;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Energy Expenditure | does energy expenditure and substrate oxidation differ between subjects randomized to the high carbohydrate versus the high protein diet? We measured resting energy expenditure with indirect calorimetry and estimated substrate oxidation with indirect calorimetry results and urine urea nitrogen excretion. We also measured total energy expenditure with doubly labeled water. | change from baseline after 4 months of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Body Composition | does body composition change more among subjects randomized to the high protein diet compared to the high carbohydrate diet? We measured body composition at baseline and again at the end of the study by DEXA - dual energy x-ray absorptiometry. | change from baseline to 4 months of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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fatty acid oxidation disorders