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
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
Inclusion_
agemax
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gender
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PhaseCountries
1EUCTR2018-000755-40-IT
(EUCTR)
17/05/201925/01/2021A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo - AMICUS ATB200-03 Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: [ATB200]
INN or Proposed INN: non applicabile
Product Name: AT2221
Product Code: [AT2221]
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
Product Name: Myozyme
Product Code: [Myozyme]
INN or Proposed INN: ALGLUCOSIDASI ALFA UMANA RICOMBINANTE
AMICUS THERAPEUTICS, INC.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Korea, Democratic People's Republic of;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden