19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | EUCTR2018-002210-12-FR (EUCTR) | 28/04/2021 | 26/10/2018 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;France;Czech Republic;Canada;Belgium;Poland;Austria;Netherlands;Germany;United Kingdom | ||
| 2 | EUCTR2018-002210-12-NO (EUCTR) | 26/03/2021 | 22/09/2020 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Norway;Germany;Netherlands | ||
| 3 | EUCTR2017-003369-85-NO (EUCTR) | 24/08/2020 | 26/06/2020 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Norway;Germany;Netherlands | ||
| 4 | EUCTR2017-003369-85-IE (EUCTR) | 18/05/2020 | 01/07/2019 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 5 | EUCTR2017-003369-85-ES (EUCTR) | 18/05/2020 | 25/05/2020 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Russian Federation;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 6 | EUCTR2018-002210-12-BE (EUCTR) | 20/03/2020 | 27/03/2020 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 7 | JPRN-JapicCTI-194731 | 30/4/2019 | 24/04/2019 | An open-label phase 3 study of lucerastat in Japanese subjects with Fabry disease | A multicenter, open-label study to determine the efficacy and safety of lucerastat oral therapy in Japanese subjects with Fabry disease | Fabry disease | Intervention name : ACT-434964 INN of the intervention : Lucerastat Dosage And administration of the intervention : 1000mg twice a day, oral administration Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | Idorsia Pharmaceuticals Japan Ltd | NULL | complete | 16 | BOTH | 20 | Phase 3 | Japan | |
| 8 | EUCTR2018-002210-12-NL (EUCTR) | 12/04/2019 | 19/11/2018 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway | ||
| 9 | EUCTR2018-002210-12-GB (EUCTR) | 18/03/2019 | 23/10/2018 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Canada;Poland;Belgium;Ireland;Austria;Australia;Netherlands;Germany;United Kingdom | ||
| 10 | EUCTR2018-002210-12-AT (EUCTR) | 19/02/2019 | 05/11/2018 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 11 | EUCTR2018-002210-12-DE (EUCTR) | 13/02/2019 | 26/10/2018 | A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease | Fabry disease MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 108 | Phase 3 | United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 12 | EUCTR2017-003369-85-NL (EUCTR) | 24/07/2018 | 16/05/2018 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway | ||
| 13 | EUCTR2017-003369-85-AT (EUCTR) | 29/06/2018 | 27/04/2018 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway | ||
| 14 | EUCTR2017-003369-85-GB (EUCTR) | 14/05/2018 | 16/04/2018 | A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease | A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY | 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] - Nutritional and Metabolic Diseases [C18] | Product Name: Lucerastat Product Code: ACT-434964 INN or Proposed INN: Lucerastat Other descriptive name: OGT923 | Idorsia Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 99 | Phase 3 | United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway |