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"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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 IncRecruiting1 Month65 YearsAll5Early Phase 1United States
2NCT04138251
(ClinicalTrials.gov)
June 20, 201920/6/2019Safety, 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 LouvainNULLRecruiting1 Year18 YearsAll5Phase 2Belgium
3EUCTR2017-004153-17-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
4NCT03871673
(ClinicalTrials.gov)
April 29, 20179/3/2019The 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 SulActive, not recruiting16 YearsN/AAll11N/ABrazil
5NCT03218904
(ClinicalTrials.gov)
March 17, 201713/7/2017Glycogen 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_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02448667
(ClinicalTrials.gov)
January 201515/5/2015Energy 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, DenmarkNULLRecruiting18 YearsN/AAll15N/ADenmark
7NCT02176096
(ClinicalTrials.gov)
July 201424/6/2014Comparison 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-GreenbergCompleted5 Years18 YearsAll4N/ACanada
8NCT02054832
(ClinicalTrials.gov)
November 201331/1/2014Sleep 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 MitchellNULLCompleted2 Years50 YearsBoth11N/ACanada
9NCT00947960
(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
10EUCTR2006-005449-12-IT
(EUCTR)
11/10/200623/05/2007EVALUATION 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 intervention As 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
INN or Proposed INN: Ramipril
Trade Name: EPHYNAL
INN or Proposed INN: Tocopherol (vit E)
Dipartimento di PediatriaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy