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 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.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
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on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02517307February 201629 July 2019Fatty 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 UniversityRecruiting18 YearsN/AAll50N/AUnited States
2NCT01461304October 20119 September 2019Compassionate Use of Triheptanoin (C7) 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 IncNot recruiting1 MonthN/AAllPhase 2United States
3NCT00840112July 20102 November 2015Vitamin 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 UniversityNot recruiting7 YearsN/ABoth1N/AUnited States