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 |
| NCT01379625 ↗ | Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder | Completed | Oregon Health and Science University | 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 | >Sponsor | >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 |
| Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | 2 |
| [disabled in preview] | 0 |
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Clinical Trial Locations for Triheptanoin
Trials by Country
Clinical Trial Progress for Triheptanoin
Clinical Trial Phase
Clinical Trial Sponsors for Triheptanoin
Sponsor Name
| Sponsor Name for Triheptanoin | |
| Sponsor | Trials |
| Ultragenyx Pharmaceutical Inc | 17 |
| University of Texas Southwestern Medical Center | 5 |
| Institut National de la Santé Et de la Recherche Médicale, France | 4 |
| [disabled in preview] | 12 |
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