257. 肝型糖原病 [臨床試験数:10,薬物数:19(DrugBank:6),標的遺伝子数:1,標的パスウェイ数:6

Searched query = "Hepatic glycogenosis", "Liver glycogenosis", "Hepatic glycogen storage disease", "Liver glycogen storage disease", "Glycogen storage disease type I", "GSDI", "von Gierke disease", "Glucose-6-phosphatase deficiency", "G6Pase deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Adult polyglucosan body disease", "Glycogen storage disease type VI", "GSDVI", "Hers disease", "Hepatic phosphorylase deficiency", "Liver phosphorylase deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
3 trials found
No.TrialIDDate_
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agemin
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PhaseCountries
1NCT03665636October 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 IncRecruiting1 Month65 YearsAll5Early Phase 1United States
2EUCTR2017-004153-17-DK03/05/201826/03/2018The 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: 20.1Level: PTClassification code 10053241Term: Glycogen storage disease type VIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: PTClassification code 10053250Term: Glycogen storage disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10053255Term: Tarui diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10016983Term: Forbes' diseaseSystem 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
3NCT00947960June 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