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

NCT01379625 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2017-03-23

Study results available
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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

Conditions

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

Interventions

DRUG

Triheptanoin

Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone

Sponsors & Collaborators

  • University of Pittsburgh

    collaborator OTHER

  • Oregon Health and Science University

    lead OTHER

Principal Investigators

  • Melanie B Gillingham, PhD · Oregon Health and Science University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-30
Primary Completion
2015-04-30
Completion
2015-04-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01379625 on ClinicalTrials.gov