256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
| 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 | EUCTR2012-002933-12-GB (EUCTR) | 07/04/2014 | 04/04/2014 | A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle Disease | A Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Sodium Valproate for GSDV Version 1.0 13th January 2014 | McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdomyolysis which can cause acute renal failure and from middle age muscle wasting and weakness. Affected patients are unable to produce lactate during ischaemic exercise due to a congenital lack of the enzyme muscle glycogen phosphorylase which is essential for glycogen metabolism. MedDRA version: 16.1;Level: LLT;Classification code 10026969;Term: McArdle's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Epilim Chrono 200 Controlled Released tablets Product Name: Epilim Chrono 200 Controlled Released tablets INN or Proposed INN: Sodium valproate Other descriptive name: Valproic acid Trade Name: Epilim Chrono 300 Controlled Released tablets Product Name: Epilim Chrono 300 Controlled Released tablets INN or Proposed INN: Sodium valproate Other descriptive name: valproic acid Trade Name: Epilim Chrono 500 Controlled Released tablets Product Name: Epilim Chrono 500 Controlled Released tablets INN or Proposed INN: Sodium valproate Other descriptive name: valproic acid | University College London | ,NULL | Not Recruiting | Female: yes Male: yes | 8 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Denmark;United Kingdom |