Trial Outcomes & Findings for Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (NCT NCT01379625)

Last Updated: 2017-03-23

Results Overview

Total energy expenditure will be measured by doubly labeled water and resting energy expenditure will be measured by indirect calorimetry at baseline and again after 4 months of either MCT or trihpetanoin treatment.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

change from baseline after 4 months of treatment

Results posted on

2017-03-23

Participant Flow

Participant milestones

Participant milestones
Measure	Medium Chain Triglyceride (MCT) Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone
Overall Study STARTED	16	16
Overall Study COMPLETED	16	16
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

There was a change in the protocol from cardiac MRI to echo after the 1st 7 participants and some of the echocardiogram results were unreadable due to technical errors. A total of 11 subjects in the MCT and 10 subjects in triheptanoin group were analyzed.

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: change from baseline after 4 months of treatment

Population: One subject in each group did not complete the doubly labeled water measures. A total of 15 in each group measured total energy expenditure at baseline and at the end of the study. This value compares the change over treatment.

Outcome measures

Outcome measures
Measure	Medium Chain Triglyceride (MCT) n=15 Participants Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin n=15 Participants Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone
Energy Expenditure	-73 kcal/day Standard Deviation 335	107 kcal/day Standard Deviation 387

PRIMARY outcome

Timeframe: 4 months

Population: Change in resting ejection fraction over 4 month treatment period calculated as: 4 month ejection fraction-baseline ejection fraction. Only participants with available data are included in this analysis.

Change in resting ejection fraction over 4 month treatment period

Outcome measures

Outcome measures
Measure	Medium Chain Triglyceride (MCT) n=11 Participants Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin n=10 Participants Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone
Ejection Fraction	-1.91 percent Standard Deviation 4.16	2.14 percent Standard Deviation 4.43

SECONDARY outcome

Timeframe: change from baseline to 4 months of treatment

Subjects will complete a submaximal treadmill exercise study at baseline. Exercise heart heart, ventilation and perceived exertion will be measured. Subjects will be randomized to MCT or triheptanoin supplementation for 4 months. At the end of treatment, the exercise test will be repeated keeping work performed constant. Change in exercise heart rate, ventilation and exertion will be compared between groups.

Outcome measures

Outcome measures
Measure	Medium Chain Triglyceride (MCT) n=16 Participants Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin n=16 Participants Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone
Exercise Heart Rate	-0.1 beats per minute Standard Deviation 9.6	-12.6 beats per minute Standard Deviation 34

Adverse Events

Medium Chain Triglyceride (MCT)

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Triheptanoin

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Medium Chain Triglyceride (MCT) n=16 participants at risk Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin n=16 participants at risk Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone
Gastrointestinal disorders Gastrointestinal Upset	75.0% 12/16 • Number of events 38 • Four months	68.8% 11/16 • Number of events 24 • Four months
Musculoskeletal and connective tissue disorders Rhabdomyolysis	25.0% 4/16 • Number of events 7 • Four months	31.2% 5/16 • Number of events 7 • Four months
Gastrointestinal disorders Diarrhea/Loose stools	37.5% 6/16 • Number of events 12 • Four months	31.2% 5/16 • Number of events 9 • Four months
Musculoskeletal and connective tissue disorders muscle pain/ elevated CPK	62.5% 10/16 • Number of events 18 • Four months	68.8% 11/16 • Number of events 16 • Four months

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

Measure	Medium Chain Triglyceride (MCT) n=16 Participants Subjects randomized to consume 20% of energy from MCT Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Triheptanoin n=16 Participants Subject randomized to consume 20% of energy from triheptanoin. Triheptanoin: Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone	Total n=32 Participants Total of all reporting groups
Age, Continuous	22.3 years STANDARD_DEVIATION 12.7 • n=16 Participants	27.2 years STANDARD_DEVIATION 15.9 • n=16 Participants	24.8 years STANDARD_DEVIATION 14.4 • n=32 Participants
Sex: Female, Male Female	10 Participants n=16 Participants	10 Participants n=16 Participants	20 Participants n=32 Participants
Sex: Female, Male Male	6 Participants n=16 Participants	6 Participants n=16 Participants	12 Participants n=32 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=16 Participants	0 Participants n=16 Participants	1 Participants n=32 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=16 Participants	16 Participants n=16 Participants	31 Participants n=32 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) Asian	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) Black or African American	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) White	16 Participants n=16 Participants	16 Participants n=16 Participants	32 Participants n=32 Participants
Race (NIH/OMB) More than one race	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=16 Participants	0 Participants n=16 Participants	0 Participants n=32 Participants
Region of Enrollment United States	16 participants n=16 Participants	16 participants n=16 Participants	32 participants n=32 Participants
Body Weight	67.1 kilograms STANDARD_DEVIATION 26.8 • n=16 Participants	62.1 kilograms STANDARD_DEVIATION 23.5 • n=16 Participants	64.6 kilograms STANDARD_DEVIATION 24.9 • n=32 Participants
Ejection Fraction	63 percent STANDARD_DEVIATION 5.3 • n=11 Participants • There was a change in the protocol from cardiac MRI to echo after the 1st 7 participants and some of the echocardiogram results were unreadable due to technical errors. A total of 11 subjects in the MCT and 10 subjects in triheptanoin group were analyzed.	57 percent STANDARD_DEVIATION 9.2 • n=10 Participants • There was a change in the protocol from cardiac MRI to echo after the 1st 7 participants and some of the echocardiogram results were unreadable due to technical errors. A total of 11 subjects in the MCT and 10 subjects in triheptanoin group were analyzed.	59.7 percent STANDARD_DEVIATION 8.0 • n=21 Participants • There was a change in the protocol from cardiac MRI to echo after the 1st 7 participants and some of the echocardiogram results were unreadable due to technical errors. A total of 11 subjects in the MCT and 10 subjects in triheptanoin group were analyzed.
Exercise Heart Rate	132 beats per minute STANDARD_DEVIATION 7 • n=16 Participants	125 beats per minute STANDARD_DEVIATION 12 • n=16 Participants	128 beats per minute STANDARD_DEVIATION 10 • n=32 Participants
Total Energy Expenditure	2567 kcal/day STANDARD_DEVIATION 604 • n=15 Participants • One participant in the MCT group and one participant in the Triheptanoin group did not return their urine samples for the doubly labeled water analysis. A total of 15 in each group were analyzed.	2230 kcal/day STANDARD_DEVIATION 512 • n=15 Participants • One participant in the MCT group and one participant in the Triheptanoin group did not return their urine samples for the doubly labeled water analysis. A total of 15 in each group were analyzed.	2392 kcal/day STANDARD_DEVIATION 572 • n=30 Participants • One participant in the MCT group and one participant in the Triheptanoin group did not return their urine samples for the doubly labeled water analysis. A total of 15 in each group were analyzed.