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"
10 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCT2021200023
08/10/202009/10/2020A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late onset GM2 gangliosidosis and ultra rare diseases within the same and similar glucosylceramidebased sphingolipid pathway Tay-Sachs Disease, Sandhoff DiseaseDrug: venglustat GZ402671
- Pharmaceutical form: tablet
- Route of administration: oral
Drug: placebo
- Pharmaceutical form: tablet
- Route of administration: oral
Tanaka TomoyukiNULLRecruiting>= 2age oldNot applicableBoth62Phase 3Spain;United States;Brazil;Russian Federation;United Kingdom;Japan
2EUCTR2019-002375-34-DE
(EUCTR)
23/07/202025/11/2019A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST Tay-Sachs diseaseSandhoff disease
MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850
MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
83Phase 3United States;Portugal;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan
3NCT04221451
(ClinicalTrials.gov)
June 29, 20206/1/2020A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid PathwayTay-Sachs Disease Sandhoff DiseaseDrug: venglustat GZ402671;Drug: placeboGenzyme, a Sanofi CompanyNULLRecruiting2 YearsN/AAll62Phase 3United States;Brazil;Germany;Japan;Russian Federation;Spain;United Kingdom
4EUCTR2019-002375-34-CZ
(EUCTR)
26/05/202026/05/2020A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST Tay-Sachs diseaseSandhoff disease
MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850
MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
83Phase 3United States;Portugal;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Argentina;Brazil;Germany;Japan
5EUCTR2019-002375-34-PT
(EUCTR)
25/05/202003/12/2019A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST Tay-Sachs diseaseSandhoff disease
MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850
MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
83Phase 3Portugal;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-002375-34-GB
(EUCTR)
23/03/202027/11/2019A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST Tay-Sachs diseaseSandhoff disease
MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850
MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
83Phase 3United States;Portugal;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan
7EUCTR2014-004995-49-GB
(EUCTR)
14/10/201508/05/2015Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients with Fabry DiseaseAn Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed with Fabry Disease Fabry disease
MedDRA version: 18.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Hormonal diseases [C19]
Product Code: SAR402671, GZ402671 or GZ/SAR402671
Other descriptive name: Genz-682452-AU
Product Code: SAR402671, GZ402671 or GZ/SAR402671
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: no
Male: yes
8Phase 2France;United States;Czech Republic;Poland;Russian Federation;United Kingdom
8EUCTR2013-005324-41-GB
(EUCTR)
07/10/201430/06/2014Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry DiseaseA Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease Fabry disease
MedDRA version: 17.0;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]
Product Code: GZ402671 / SAR402671
Other descriptive name: Genz-682452-AA
Product Code: GZ402671 / SAR402671
Other descriptive name: Genz-682452-AA
Genzyme CorporationNULLNot RecruitingFemale: no
Male: yes
8Phase 2France;United States;Czech Republic;Poland;Russian Federation;United Kingdom
9EUCTR2013-005324-41-CZ
(EUCTR)
19/09/201403/07/2014Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry DiseaseA Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease Fabry disease
MedDRA version: 17.1;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]
Product Code: GZ402671 / SAR402671
Other descriptive name: Genz-682452-AA
Product Code: GZ402671 / SAR402671
Other descriptive name: Genz-682452-AA
Genzyme CorporationNULLNot RecruitingFemale: no
Male: yes
8Phase 2France;United States;Czech Republic;Poland;Russian Federation;United Kingdom
10EUCTR2019-002375-34-AT
(EUCTR)
06/12/2019A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST Tay-Sachs diseaseSandhoff disease
MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850
MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNAFemale: yes
Male: yes
83Phase 3United States;Portugal;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Argentina;Brazil;Germany;Japan