CLINICAL TRIALS PROFILE FOR TRIHEPTANOIN

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

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
Glut1 Deficiency Syndrome 1, Autosomal Recessive	2

Condition MeSH

Condition MeSH for TRIHEPTANOIN
Intervention	Trials
Syndrome	12
Carbohydrate Metabolism, Inborn Errors	11
Metabolic Diseases	5
Glycogen Storage Disease	5

Clinical Trial Locations for TRIHEPTANOIN

Trials by Country

Trials by Country for TRIHEPTANOIN
Location	Trials
United States	49
France	7
United Kingdom	6
Spain	6
Poland	3

Trials by US State

Trials by US State for TRIHEPTANOIN
Location	Trials
Texas	10
Pennsylvania	7
Florida	5
Washington	3
New York	3

Clinical Trial Progress for TRIHEPTANOIN

Clinical Trial Phase

Clinical Trial Phase for TRIHEPTANOIN
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	18
Phase 1/Phase 2	4

Clinical Trial Status

Clinical Trial Status for TRIHEPTANOIN
Clinical Trial Phase	Trials
Completed	15
Recruiting	5
No longer available	4

Clinical Trial Sponsors for TRIHEPTANOIN

Sponsor Name

Sponsor Name for TRIHEPTANOIN
Sponsor	Trials
Ultragenyx Pharmaceutical Inc	19
University of Texas Southwestern Medical Center	5
Juan Pascual	4

Sponsor Type

Sponsor Type for TRIHEPTANOIN
Sponsor	Trials
Other	41
Industry	19
NIH	3