257. 肝型糖原病 Hepatic glycogenosis Clinical trials / Disease details
臨床試験数 : 14 / 薬物数 : 28 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8
| 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 | Completed | 1 Month | 65 Years | All | 4 | 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 |