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Last Updated: October 22, 2020

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CLINICAL TRIALS PROFILE FOR TRIHEPTANOIN

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All Clinical Trials for Triheptanoin

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00328159 Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids Completed Assistance Publique - Hôpitaux de Paris N/A 2006-06-01 Usual dietary therapies of mitochondrial fatty acid oxidation disorders (FAO) are based on 3 strategies: - limitation of lipid intake in the diet; - supplementation of the diet with medium-chain triglycerides (MCT) for patients affected with disorders of long-chain FAO; - some specific supplementations (for example, L-carnitine). These strategies are often ineffective. The aim of the present study is to evaluate new therapeutic ways based on the underlying energetic defect observed in these disorders. The long-term goal is to develop efficient therapies of these disorders.
NCT00947960 Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease Completed Ultragenyx Pharmaceutical Inc Phase 2 2009-06-01 The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.
NCT00947960 Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease Completed Baylor Research Institute Phase 2 2009-06-01 The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.
NCT00983788 Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects Completed Groupe Hospitalier Pitie-Salpetriere Phase 2 2009-10-01 The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized, double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers: Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after infusion of stable isotopes (palmitate and glucose tracers).
NCT00983788 Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects Completed Rigshospitalet, Denmark Phase 2 2009-10-01 The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized, double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers: Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after infusion of stable isotopes (palmitate and glucose tracers).
NCT01379625 Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder Completed University of Pittsburgh Phase 2 2011-09-01 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
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Triheptanoin

Condition Name

Condition Name for Triheptanoin
Intervention Trials
Glucose Transporter Type 1 Deficiency Syndrome 7
Glut1 Deficiency Syndrome 4
GLUT1DS1 3
Epilepsy 2
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Condition MeSH

Condition MeSH for Triheptanoin
Intervention Trials
Syndrome 12
Carbohydrate Metabolism, Inborn Errors 11
Metabolic Diseases 5
Glycogen Storage Disease 5
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Clinical Trial Locations for Triheptanoin

Trials by Country

Trials by Country for Triheptanoin
Location Trials
United States 39
France 6
United Kingdom 3
Denmark 2
Spain 2
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Trials by US State

Trials by US State for Triheptanoin
Location Trials
Texas 8
Pennsylvania 6
Massachusetts 3
Illinois 3
Florida 3
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Clinical Trial Progress for Triheptanoin

Clinical Trial Phase

Clinical Trial Phase for Triheptanoin
Clinical Trial Phase Trials
Phase 3 2
Phase 2 16
Phase 1/Phase 2 3
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Clinical Trial Status

Clinical Trial Status for Triheptanoin
Clinical Trial Phase Trials
Recruiting 8
Not yet recruiting 7
Completed 5
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Clinical Trial Sponsors for Triheptanoin

Sponsor Name

Sponsor Name for Triheptanoin
Sponsor Trials
Ultragenyx Pharmaceutical Inc 15
University of Texas Southwestern Medical Center 5
Institut National de la Santé Et de la Recherche Médicale, France 4
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Sponsor Type

Sponsor Type for Triheptanoin
Sponsor Trials
Other 32
Industry 15
NIH 3
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