Phase 2 Completed N=32 Randomized Double-blind Treatment

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency · Carnitine Palmitoyltransferase 2 (CPT2) Deficiency · Mitochondrial Trifunctional Protein (TFP) Deficiency · Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency

Source: ClinicalTrials.gov NCT01379625 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Mar 2017

Primary outcomePrimary: Energy Expenditure — -73; 107 kcal/day

Summary

Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy. They sometimes develop muscle breakdown, and severe pain with exercise or illness. They can also develop a heart that does not function properly. These patients are tired and expend less energy than people who do not have a long-chain fatty acid oxidation disorder. However, they can use a supplement oil called medium chain triglyceride or MCT. This study will determine if a new experimental oil called Triheptanoin can decrease the muscle pain and increase the heart function and the amount of energy in patients with long-chain fatty acid oxidation disorders. Funding source - FDA's OOPD

Outcome Measures

Outcome	Result	p-value
PRIMARY Energy Expenditure	-73; 107	—
PRIMARY Ejection Fraction	-1.91; 2.14	—
SECONDARY Exercise Heart Rate	-0.1; -12.6	—

Eligibility Criteria

Inclusion Criteria

Confirmed diagnosis of Very long-chain acylCoA dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, or Long-chain 3 hydroxyacylCoA dehydrogenase (LCHAD) deficiency
> 7 years
Ability to travel to CRC to participate
Ability to follow protocol

Exclusion Criteria

Hgb < 10 g/dl
Peripheral neuropathy that limits ability to complete treadmill studies
Inclusion in another research study that alters macronutrient intake
Pregnant females
history of myocardial infarction or cardiovascular disease

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Data sourced from ClinicalTrials.gov (NCT01379625). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.