Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Conditions
Keywords
fatty acid oxidation disorders
Brief summary
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
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.
Interventions
BEHAVIORALDiet counseling
Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home.
Sponsors
Oregon Health and Science University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 45 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Energy Expenditure | change from baseline after 4 months of treatment | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Body Composition | change from baseline to 4 months of treatment | 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. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| High Carbohydrate Diet 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.
Diet counseling: Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home. | 6 |
| High Protein Diet 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.
Diet counseling: Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home. | 7 |
| Total | 13 |
Baseline characteristics
| Characteristic | High Protein Diet | Total | High Carbohydrate Diet |
|---|---|---|---|
| Age, Continuous | 17 years STANDARD_DEVIATION 10 | 16 years STANDARD_DEVIATION 8 | 14 years STANDARD_DEVIATION 5 |
| Region of Enrollment North America | 6 participants | 12 participants | 6 participants |
| Region of Enrollment Sweden | 1 participants | 1 participants | 0 participants |
| Sex: Female, Male Female | 4 Participants | 6 Participants | 2 Participants |
| Sex: Female, Male Male | 3 Participants | 7 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 7 |
| other Total, other adverse events | 0 / 6 | 0 / 7 |
| serious Total, serious adverse events | 0 / 6 | 0 / 7 |
Outcome results
Primary
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.
Time frame: change from baseline after 4 months of treatment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| High Carbohydrate Diet | Energy Expenditure | 2045 kcal | Standard Deviation 273 |
| High Protein Diet | Energy Expenditure | 2343 kcal | Standard Deviation 507 |
Secondary
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.
Time frame: change from baseline to 4 months of treatment
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| High Carbohydrate Diet | Body Composition | Lean Mass | -0.75 change in kg | Standard Deviation 1.34 |
| High Carbohydrate Diet | Body Composition | Fat Mass | 0.51 change in kg | Standard Deviation 4.34 |
| High Protein Diet | Body Composition | Lean Mass | 0.78 change in kg | Standard Deviation 1.17 |
| High Protein Diet | Body Composition | Fat Mass | 0.43 change in kg | Standard Deviation 0.9 |