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 257. 肝型糖原病　［臨床試験数：10，薬物数：15（DrugBank：4），標的遺伝子数：1，標的パスウェイ数：4］ 

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 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"
No.TrialIDDate_
enrollement		Last_Refreshed_
on		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03665636December 201930 September 2019Anaplerotic 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 IncNot recruiting1 Month65 YearsAll5Early Phase 1United States
2NCT04138251June 20, 20194 November 2019Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 DeficiencyEvaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 DeficiencyGlycogen Storage Disease Type I;Glucose 6 Phosphatase DeficiencyDrug: EmpagliflozinCliniques universitaires Saint-Luc- Université Catholique de LouvainRecruiting1 Year18 YearsAll5Phase 2Belgium
3EUCTR2017-004153-17-DK03/05/201814 May 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 GlycogenosesCori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also 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 Level: PT 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 System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: LLT 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
Pharmaceutical Form: Oral liquid
INN or Proposed INN: Not available
CAS Number: 210-647-2
Current Sponsor code: UX0007
Other descriptive name: TRIHEPTANOIN
Concentration unit: % percent
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral liquid
Route of administration of the placebo: Oral use		Copenhagen Neuromuscular CenterAuthorisedFemale: yes
Male: yes		20Phase 2Denmark
4NCT03871673April 29, 20179 September 2019The Use of Uncooked Sweet Polvilho to Treat Hepatic Glycogen Storage DiseasesA Novel Approach to Treatment of Hepatic Glycogen Storage Diseases: a Study Based on the Use of Uncooked Sweet PolvilhoGlycogen Storage Disease Type IDietary Supplement: sweet polvilho;Dietary Supplement: CornstarchHospital de Clinicas de Porto AlegreConselho Nacional de Desenvolvimento Científico e Tecnológico;Fundação Médica do Rio Grande do SulNot recruiting16 YearsN/AAll11N/ABrazil
5NCT03218904March 17, 20172 September 2019Glycogen Storage Disease Breath Test StudyApplication of Novel Techniques to Devise Nutritional Therapies in Subjects With Glycogen Storage Disease Type IPatient Compliance;HealthyDietary Supplement: Glucose intake;Dietary Supplement: Carbohydrates intakeUniversity of British ColumbiaSaudi Arabian Cultural Bureau, Ottawa;Canadian Institutes of Health Research (CIHR)Recruiting5 Years35 YearsAll38N/ACanada
No.TrialIDDate_
enrollement		Last_Refreshed_
on		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT02448667January 201516 December 2017Energy Supplements to Improve Exercise Tolerance in Metabolic MyopathiesEnergy Supplements to Improve Exercise Tolerance in Metabolic MyopathiesGlycogen Storage Disease Type IIIDietary Supplement: FAXE Kondi;Dietary Supplement: Faxe Kondi FreeRigshospitalet, DenmarkRecruiting18 YearsN/AAll15N/ADenmark
7NCT02176096July 201416 December 2017Comparison of the Effect of a Novel Starch (Glycosade) Versus Gastrostomy Tube-Dextrose Infusion on Overnight Euglycaemia Control in Children With Glycogen Storage Disease Type I: Open Label Demonstration TrialComparison of the Effect of a Novel Starch (Glycosade) Versus Gastrostomy Tube-Dextrose Infusion on Overnight Euglycaemia Control in Children With Glycogen Storage Disease Type I: Open Label Demonstration TrialGlycogen Storage Disorder Type 1;Hypoglycemia;Cornstarch;GlycosadeDietary Supplement: GlycosadeUniversity of ManitobaCo-Investigator - Dr. Cheryl Rockman-GreenbergNot recruiting5 Years18 YearsAll4N/ACanada
8NCT02054832November 20136 October 2015Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked CornstarchA Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked CornstarchGlycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0Dietary Supplement: GlycosadeJohn MitchellNot recruiting2 Years50 YearsBoth11N/ACanada
9NCT00947960June 200911 June 2018Triheptanoin 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 IncNot recruiting18 Years75 YearsAll23Phase 2France;United States
10EUCTR2006-005449-12-IT11/10/20063 April 2012EVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b - GSD1: study of specific therapeutic interventionEVALUATION OF THE EFFICACY OF THE TREATMENT WITH ACE-INHIBITORS ON THE RENAL DAMAGE IN PATIENTS AFFECTED BY GLYCOGEN STORAGE DISEASE TYPE 1 AND OF THE VITAMINE E ON NEUTROPENIA OF PATIENTS WITH GSD1b - GSD1: study of specific therapeutic interventionAs concern objective number 1: Patients affected by glycogen storage disease (GSD) type 1a and patients affected by GSD1b with renal dysfunction including glomerular hyperfiltration, microalbuminuria and/or proteinuria As concern objective number 2: Patients affected by GSD1b showing neutropenia
MedDRA version: 9.1 Level: LLT Classification code 10018464 Term: Glycogen storage disease type I		Trade Name: QUARK
Pharmaceutical Form: Tablet
INN or Proposed INN: Ramipril
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Trade Name: EPHYNAL
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Tocopherol (vit E)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-		Dipartimento di PediatriaAuthorisedFemale: yes
Male: yes		Italy

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