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

  
1 trial found
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agemin
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agemax
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PhaseCountries
1NCT01461304
(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