19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-004689-32-FI
(EUCTR)
12/10/202117/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease REPLAGAL is intended for use for patients with Fabry disease.
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Brazil;Belgium;Poland;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina
2EUCTR2018-004689-32-PL
(EUCTR)
08/10/202107/10/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease Fabry disease
MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Australia;Latvia;Germany;Sweden;Bosnia and Herzegovina
3EUCTR2018-004689-32-GR
(EUCTR)
06/10/202106/10/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease REPLAGAL is intended for use for patients with Fabry disease.
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina
4EUCTR2018-004689-32-PT
(EUCTR)
24/09/202111/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease Fabry disease
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina
5EUCTR2018-004689-32-SE
(EUCTR)
24/08/202117/08/2021Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry DiseaseA Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease Fabry disease
MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: REPLAGAL ®
Product Name: REPLAGAL
Product Code: SHP675
INN or Proposed INN: AGALSIDASE ALFA
Other descriptive name: Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina