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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03665636 (ClinicalTrials.gov) | October 16, 2020 | 23/8/2018 | Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I | Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I | Glycogen Storage Disease Type I | Drug: Triheptanoin | Areeg El-Gharbawy | Ultragenyx Pharmaceutical Inc | Recruiting | 1 Month | 65 Years | All | 5 | Early Phase 1 | United States |
| 2 | NCT04138251 (ClinicalTrials.gov) | June 20, 2019 | 20/6/2019 | Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency | Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 Deficiency | Glycogen Storage Disease Type I;Glucose 6 Phosphatase Deficiency | Drug: Empagliflozin | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | NULL | Recruiting | 1 Year | 18 Years | All | 5 | Phase 2 | Belgium |
| 3 | EUCTR2017-004153-17-DK (EUCTR) | 03/05/2018 | 26/03/2018 | The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses | Evaluation 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 Center | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Denmark | ||
| 4 | NCT03871673 (ClinicalTrials.gov) | April 29, 2017 | 9/3/2019 | The Use of Uncooked Sweet Polvilho to Treat Hepatic Glycogen Storage Diseases | A Novel Approach to Treatment of Hepatic Glycogen Storage Diseases: a Study Based on the Use of Uncooked Sweet Polvilho | Glycogen Storage Disease Type I | Dietary Supplement: sweet polvilho;Dietary Supplement: Cornstarch | Hospital de Clinicas de Porto Alegre | Conselho Nacional de Desenvolvimento Científico e Tecnológico;Fundação Médica do Rio Grande do Sul | Active, not recruiting | 16 Years | N/A | All | 11 | N/A | Brazil |
| 5 | NCT03218904 (ClinicalTrials.gov) | March 17, 2017 | 13/7/2017 | Glycogen Storage Disease Breath Test Study | Application of Novel Techniques to Devise Nutritional Therapies in Subjects With Glycogen Storage Disease Type I | Patient Compliance;Healthy | Dietary Supplement: Glucose intake;Dietary Supplement: Carbohydrates intake | University of British Columbia | Saudi Arabian Cultural Bureau, Ottawa;Canadian Institutes of Health Research (CIHR) | Recruiting | 5 Years | 35 Years | All | 38 | N/A | Canada |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02448667 (ClinicalTrials.gov) | January 2015 | 15/5/2015 | Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies | Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies | Glycogen Storage Disease Type III | Dietary Supplement: FAXE Kondi;Dietary Supplement: Faxe Kondi Free | Rigshospitalet, Denmark | NULL | Recruiting | 18 Years | N/A | All | 15 | N/A | Denmark |
| 7 | NCT02176096 (ClinicalTrials.gov) | July 2014 | 24/6/2014 | Comparison 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 Trial | Comparison 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 Trial | Glycogen Storage Disorder Type 1;Hypoglycemia;Cornstarch;Glycosade | Dietary Supplement: Glycosade | University of Manitoba | Co-Investigator - Dr. Cheryl Rockman-Greenberg | Completed | 5 Years | 18 Years | All | 4 | N/A | Canada |
| 8 | NCT02054832 (ClinicalTrials.gov) | November 2013 | 31/1/2014 | Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch | A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch | Glycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0 | Dietary Supplement: Glycosade | John Mitchell | NULL | Completed | 2 Years | 50 Years | Both | 11 | N/A | Canada |
| 9 | NCT00947960 (ClinicalTrials.gov) | June 2009 | 24/7/2009 | Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease | A Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled Study | Adult Polyglucosan Body Disease;Glycogen Brancher Enzyme Deficiency;Glycogen Storage Disease Type IV | Drug: Triheptanoin;Other: Vegetable Oil | Baylor Research Institute | Ultragenyx Pharmaceutical Inc | Completed | 18 Years | 75 Years | All | 23 | Phase 2 | France;United States |
| 10 | EUCTR2006-005449-12-IT (EUCTR) | 11/10/2006 | 23/05/2007 | EVALUATION 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 | EVALUATION 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 Pediatria | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy |