317. 三頭酵素欠損症 Trifunctional protein deficiency Clinical trials / Disease details

臨床試験数 : 4 / 薬物数 : 9 - (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	NCT05411835 (ClinicalTrials.gov)	August 1, 2022	27/5/2022	Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders	Safety and Tolerability of Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders	Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;Carnitine Palmitoyltransferase Deficiency 2;Very Long Chain Acyl Coa Dehydrogenase Deficiency;Trifunctional Protein Deficiency	Dietary Supplement: Nutritional Ketone Supplement;Dietary Supplement: Isocaloric Placebo Supplement	Oregon Health and Science University	NULL	Recruiting	18 Years	N/A	All	5	Early Phase 1	United States
2	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
3	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
4	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