19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
16 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-003369-85-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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Norway;Germany;Netherlands
2EUCTR2017-003369-85-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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
3EUCTR2017-003369-85-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
4EUCTR2018-002210-12-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;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
5JPRN-JapicCTI-194975
01/10/201927/09/2019An multicenter open-label long-term continuous administration study to investigate the safety and tolerability of lucerastat in Japanese Fabry disease patientsAn multicenter open-label long-term continuous administration study to investigate the safety and tolerability of lucerastat in Japanese Fabry disease patients Fabry diseaseIntervention name : Lucerastat
INN of the intervention : lucerastat
Dosage And administration of the intervention : 1000 mg twice daily
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
Idorsia Pharmaceuticals Japan LtdNULLother16BOTH20Phase 3Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6JPRN-JapicCTI-194731
30/4/201924/04/2019An open-label phase 3 study of lucerastat in Japanese subjects with Fabry diseaseA multicenter, open-label study to determine the efficacy and safety of lucerastat oral therapy in Japanese subjects with Fabry disease Fabry diseaseIntervention 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 LtdNULLcomplete16BOTH20Phase 3Japan
7EUCTR2018-002210-12-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: 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;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway
8EUCTR2018-002210-12-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
9EUCTR2018-002210-12-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]
Idorsia Pharmaceuticals LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
108Phase 3France;United States;Czech Republic;Canada;Poland;Australia;Austria;Netherlands;Germany;United Kingdom
10EUCTR2018-002210-12-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;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03737214
(ClinicalTrials.gov)
December 18, 20186/11/2018A Study to Evaluate the Long-term Safety and Tolerability of 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 DiseaseFabry DiseaseDrug: LucerastatIdorsia Pharmaceuticals Ltd.NULLRecruiting18 YearsN/AAll108Phase 3United States;Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;Czechia;France
12EUCTR2017-003369-85-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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Netherlands;Germany;Norway
13EUCTR2017-003369-85-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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
99Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Belgium;Poland;Australia;Germany;Netherlands;Norway
14NCT03425539
(ClinicalTrials.gov)
June 21, 201816/1/2018Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects 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 DiseaseFabry DiseaseDrug: Lucerastat;Drug: PlaceboIdorsia Pharmaceuticals Ltd.NULLRecruiting18 YearsN/AAll99Phase 3United States;Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;Czechia;France
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT02930655
(ClinicalTrials.gov)
February 1, 201510/10/2016A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry DiseaseA Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement TherapyFabry DiseaseDrug: Lucerastat;Drug: Enzyme replacement therapy (ERT)Idorsia Pharmaceuticals Ltd.NULLCompleted18 YearsN/AAll14Phase 1Germany