317. Trifunctional protein deficiency Clinical trials / Disease details


Clinical trials : 4 Drugs : 9 - (DrugBank : 4) / Drug target gene : 1 - Drug target pathway : 1

  
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PhaseCountries
1NCT05411835
(ClinicalTrials.gov)
August 1, 202227/5/2022Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation DisordersSafety and Tolerability of Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation DisordersLong-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;Carnitine Palmitoyltransferase Deficiency 2;Very Long Chain Acyl Coa Dehydrogenase Deficiency;Trifunctional Protein DeficiencyDietary Supplement: Nutritional Ketone Supplement;Dietary Supplement: Isocaloric Placebo SupplementOregon Health and Science UniversityNULLRecruiting18 YearsN/AAll5Early Phase 1United States
2NCT02517307
(ClinicalTrials.gov)
February 20162/7/2015Fatty Acid Oxidation Defects and Insulin SensitivityRole of Fatty Acid Oxidation Defects in Insulin SensitivityVery 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, MyopathicDrug: Intralipid/Heparin;Drug: Glycerol/Saline;Drug: Hyperinsulinemic euglycemic clampOregon Health and Science UniversityNULLCompleted18 YearsN/AAll52N/AUnited States
3NCT00840112
(ClinicalTrials.gov)
July 20109/2/2009Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated NeuropathyVitamin E Treatment for LCHAD Associated NeuropathyPeripheral Neuropathy;Mitochondrial Trifunctional Protein DeficiencyDietary Supplement: Vitamin E supplementOregon Health and Science UniversityNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Oregon State UniversityTerminated7 YearsN/ABoth1N/AUnited States
4NCT01461304
(ClinicalTrials.gov)
April 17, 200925/10/2011Dietary Therapy for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery 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 SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer available1 MonthN/AAllUnited States