317. 三頭酵素欠損症 Trifunctional protein deficiency Clinical trials / Disease details
臨床試験数 : 3 / 薬物数 : 8 - (DrugBank : 4) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02517307 (ClinicalTrials.gov) | February 2016 | 2/7/2015 | Fatty Acid Oxidation Defects and Insulin Sensitivity | Role of Fatty Acid Oxidation Defects in Insulin Sensitivity | Very Long-chain Acyl-CoA Dehydrogenase Deficiency;Trifunctional Protein Deficiency;Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;Medium-chain Acyl-CoA Dehydrogenase Deficiency;Normal Volunteers;Carnitine Palmitoyltransferase II Deficiency, Myopathic | Drug: Intralipid/Heparin;Drug: Glycerol/Saline;Drug: Hyperinsulinemic euglycemic clamp | Oregon Health and Science University | NULL | Completed | 18 Years | N/A | All | 52 | N/A | United States |
| 2 | NCT00840112 (ClinicalTrials.gov) | July 2010 | 9/2/2009 | Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy | Vitamin E Treatment for LCHAD Associated Neuropathy | Peripheral Neuropathy;Mitochondrial Trifunctional Protein Deficiency | Dietary Supplement: Vitamin E supplement | Oregon Health and Science University | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Oregon State University | Terminated | 7 Years | N/A | Both | 1 | N/A | United States |
| 3 | NCT01461304 (ClinicalTrials.gov) | April 17, 2009 | 25/10/2011 | Dietary Therapy for Inherited Disorders of Energy Metabolism | Dietary Therapy for Inherited Disorders of Energy Metabolism | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth Syndrome | Drug: triheptanoin | Jerry Vockley, MD, PhD | Ultragenyx Pharmaceutical Inc | No longer available | 1 Month | N/A | All | United States |