19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2016-000378-38-FR (EUCTR) | 13/05/2019 | 15/05/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Norway;Netherlands;Germany | ||
| 2 | EUCTR2016-000378-38-FI (EUCTR) | 12/03/2019 | 06/02/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 78 | Phase 3 | United States;Czechia;Slovenia;Finland;Spain;Turkey;United Kingdom;Switzerland;Italy;France;Hungary;Canada;Argentina;Brazil;Belgium;Australia;Denmark;Norway;Germany;Netherlands;Sweden | ||
| 3 | EUCTR2016-001318-11-NL (EUCTR) | 27/03/2018 | 17/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom | ||
| 4 | EUCTR2016-001318-11-DE (EUCTR) | 15/12/2017 | 10/01/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 5 | EUCTR2016-000378-38-IT (EUCTR) | 20/11/2017 | 06/02/2018 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta - Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | PROTALIX LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;Italy;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway | ||
| 6 | EUCTR2016-001318-11-SI (EUCTR) | 13/11/2017 | 05/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 7 | EUCTR2016-000378-38-SI (EUCTR) | 14/08/2017 | 30/06/2017 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany | ||
| 8 | EUCTR2016-000378-38-NL (EUCTR) | 24/05/2017 | 17/01/2017 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 9 | EUCTR2016-001318-11-GB (EUCTR) | 24/03/2017 | 04/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 10 | EUCTR2016-001318-11-CZ (EUCTR) | 19/01/2017 | 12/12/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 11 | EUCTR2016-001318-11-ES (EUCTR) | 11/01/2017 | 24/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | Phase 3 | Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 12 | EUCTR2016-000378-38-BE (EUCTR) | 05/12/2016 | 30/09/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 13 | EUCTR2016-000378-38-HU (EUCTR) | 15/09/2016 | 11/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Germany;Netherlands;Norway | ||
| 14 | EUCTR2016-000378-38-GB (EUCTR) | 07/09/2016 | 08/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 15 | EUCTR2016-000378-38-ES (EUCTR) | 22/08/2016 | 15/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway |