257. 肝型糖原病 Hepatic glycogenosis Clinical trials / Disease details


臨床試験数 : 14 薬物数 : 27 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8

  
3 trials found
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1NCT03665636
(ClinicalTrials.gov)
October 16, 202023/8/2018Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type IAnaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type IGlycogen Storage Disease Type IDrug: TriheptanoinAreeg El-GharbawyUltragenyx Pharmaceutical IncCompleted1 Month65 YearsAll4Early Phase 1United States
2EUCTR2017-004153-17-DK
(EUCTR)
03/05/201826/03/2018The effect of Triheptanoin on excercise in adults and adolescence with glycogenosesEvaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV.
MedDRA version: 20.1;Level: PT;Classification code 10053241;Term: Glycogen storage disease type VII;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10053255;Term: Tarui disease;Classification code 10016983;Term: Forbes' disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Copenhagen Neuromuscular CenterNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Denmark
3NCT00947960
(ClinicalTrials.gov)
June 200924/7/2009Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body DiseaseA Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled StudyAdult Polyglucosan Body Disease;Glycogen Brancher Enzyme Deficiency;Glycogen Storage Disease Type IVDrug: Triheptanoin;Other: Vegetable OilBaylor Research InstituteUltragenyx Pharmaceutical IncCompleted18 Years75 YearsAll23Phase 2France;United States