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 | 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 | ||
| 3 | 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 |