317. 三頭酵素欠損症 [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]
Searched query = "Trifunctional protein deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | NCT02517307 | February 2016 | 29 July 2019 | 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 | Recruiting | 18 Years | N/A | All | 50 | N/A | United States | |
2 | NCT01461304 | October 2011 | 9 September 2019 | Compassionate Use of Triheptanoin (C7) 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 | Not recruiting | 1 Month | N/A | All | Phase 2 | United States | |
3 | NCT00840112 | July 2010 | 2 November 2015 | 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 | Not recruiting | 7 Years | N/A | Both | 1 | N/A | United States |