Trial Outcomes & Findings for High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders (NCT NCT01494051)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

13 participants

Primary outcome timeframe

change from baseline after 4 months of treatment

Results posted on

2020-04-29

Participant Flow

Participant milestones

Participant milestones
Measure	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.	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.
Overall Study STARTED	6	7
Overall Study COMPLETED	6	7
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	High Carbohydrate Diet n=6 Participants 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.	High Protein Diet n=7 Participants 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.	Total n=13 Participants Total of all reporting groups
Age, Continuous	14 years STANDARD_DEVIATION 5 • n=5 Participants	17 years STANDARD_DEVIATION 10 • n=7 Participants	16 years STANDARD_DEVIATION 8 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Region of Enrollment North America	6 participants n=5 Participants	6 participants n=7 Participants	12 participants n=5 Participants
Region of Enrollment Sweden	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	High Carbohydrate Diet n=6 Participants 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.	High Protein Diet n=7 Participants 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.
Energy Expenditure	2045 kcal Standard Deviation 273	2343 kcal Standard Deviation 507

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	High Carbohydrate Diet n=6 Participants 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.	High Protein Diet n=7 Participants 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.
Body Composition Lean Mass	-0.75 change in kg Standard Deviation 1.34	0.78 change in kg Standard Deviation 1.17
Body Composition Fat Mass	0.51 change in kg Standard Deviation 4.34	0.43 change in kg Standard Deviation 0.9

Adverse Events

High Carbohydrate Diet

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

High Protein Diet

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Melanie Gillingham

Oregon Health & Science University

Phone: 503-494-1682

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place