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

  
15 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-002210-12-FR
(EUCTR)
28/04/202126/10/2018A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;France;Czech Republic;Canada;Belgium;Poland;Austria;Netherlands;Germany;United Kingdom
2EUCTR2018-002210-12-NO
(EUCTR)
26/03/202122/09/2020A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Norway;Germany;Netherlands
3EUCTR2018-002210-12-IT
(EUCTR)
22/10/202017/06/2021A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 - Not applicable 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;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 LTDNULLNot RecruitingFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
4EUCTR2017-003369-85-NO
(EUCTR)
24/08/202026/06/2020A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLNot RecruitingFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Norway;Germany;Netherlands
5EUCTR2017-003369-85-IT
(EUCTR)
16/07/202007/10/2020A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;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 LTDNULLNot RecruitingFemale: yes
Male: yes
108Phase 3United States;Finland;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
6EUCTR2017-003369-85-IE
(EUCTR)
18/05/202001/07/2019A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLNot RecruitingFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
7EUCTR2017-003369-85-ES
(EUCTR)
18/05/202025/05/2020A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Russian Federation;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
8EUCTR2018-002210-12-BE
(EUCTR)
20/03/202027/03/2020A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
9EUCTR2018-002210-12-NL
(EUCTR)
12/04/201919/11/2018A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway
10EUCTR2018-002210-12-GB
(EUCTR)
18/03/201923/10/2018A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Canada;Poland;Belgium;Ireland;Austria;Australia;Netherlands;Germany;United Kingdom
11EUCTR2018-002210-12-AT
(EUCTR)
19/02/201905/11/2018A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
12EUCTR2018-002210-12-DE
(EUCTR)
13/02/201926/10/2018A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3United States;Spain;Ireland;Austria;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
13EUCTR2017-003369-85-NL
(EUCTR)
24/07/201816/05/2018A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLNot RecruitingFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway
14EUCTR2017-003369-85-AT
(EUCTR)
29/06/201827/04/2018A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLNot RecruitingFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
15EUCTR2017-003369-85-GB
(EUCTR)
14/05/201816/04/2018A research study to study the effects of a new oral drug called lucerastat in adults with Fabry diseaseA 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway