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

  
1 trial found
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1EUCTR2012-002933-12-GB
(EUCTR)
07/04/201404/04/2014A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle DiseaseA 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,NULLNot 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): noUnited States;Denmark;United Kingdom