19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2021-005402-10-DE
(EUCTR)
23/03/202205/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
2Phase 3Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan
2EUCTR2021-005402-10-FR
(EUCTR)
27/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLNAFemale: yes
Male: yes
50Phase 3United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan