Phase 2
N=32
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
Bottom Line
View on ClinicalTrials.gov: NCT01379625 ↗Enrolled (actual)
32
Serious AEs
0.0%
Results posted
Mar 2017
Primary outcome: Primary: Energy Expenditure — -73; 107 kcal/day
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Triheptanoin (Drug)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- All
- Sponsor
- Oregon Health and Science University
- Primary completion
- Apr 2015
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 | — |
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
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
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.