256. Muscle glycogenosis Clinical trials / Disease details
Clinical trials : 180 / Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134
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 |
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1 | EUCTR2018-000755-40-IT (EUCTR) | 17/05/2019 | 25/01/2021 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 |