Gz402671    (DrugBank: -)

2 diseases
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease10
67Polycystic kidney disease10

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.
10 / 784 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

67. Polycystic kidney disease    [ 186 clinical trials,   196 drugs,   (DrugBank: 47 drugs),   35 drug target genes,   146 drug target pathways]
Searched query = "Polycystic kidney disease", "PKD", "PCKD", "Polycystic kidney", "ADPKD", "ARPKD"
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.
10 / 186 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-004084-12-AT
(EUCTR)
11/04/201914/01/2019A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of;United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada
2JPRN-JapicCTI-184192
31/1/201905/11/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients.Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) Polycystic kidney, autosomal dominantIntervention name : GZ402671
INN of the intervention : venglustat
Dosage And administration of the intervention : once daily for 24 months
Control intervention name : Placebo
INN of the control intervention : -
Dosage And administration of the control intervention : once daily for 24 months
Sanofi K.K.NULLrecruiting1855BOTH640Japan, Asia except Japan, North America, South America, Europe, Oceania
3EUCTR2017-004084-12-GB
(EUCTR)
28/01/201924/05/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Austria;Switzerland;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of
4EUCTR2017-004084-12-DK
(EUCTR)
07/11/201823/05/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Denmark;Australia;Germany;Netherlands;China;Japan;Korea, Republic of
5NCT03687554
(ClinicalTrials.gov)
October 5, 201826/9/2018Effect of Venglustat in Patients With Renal ImpairmentA Phase I, Single-Center, Open-label, Single Dose Pharmacokinetic and Tolerability Study of GZ402671 in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal FunctionHealthy Volunteers;Polycystic Kidney, Autosomal DominantDrug: Venglustat GZ/SAR402671Genzyme, a Sanofi CompanyNULLCompleted18 Years79 YearsAll24Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03523728
(ClinicalTrials.gov)
October 4, 20181/5/2018A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)Polycystic Kidney, Autosomal DominantDrug: Venglustat GZ402671;Drug: PlaceboGenzyme, a Sanofi CompanyNULLRecruiting18 Years55 YearsAll640Phase 2;Phase 3United States;Argentina;Australia;Austria;Belgium;Canada;China;Czechia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Romania;Spain;Taiwan;United Kingdom
7EUCTR2017-004084-12-BE
(EUCTR)
31/08/201831/05/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of
8EUCTR2017-004084-12-PT
(EUCTR)
06/08/201811/06/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of
9EUCTR2017-004084-12-DE
(EUCTR)
10/07/201822/05/2018A Medical Research Study Designed to Determine if Venglustat can be aFuture Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of
10EUCTR2017-004084-12-CZ
(EUCTR)
03/07/201813/06/2018A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD PatientsMulticenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - STAGED-PKD Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of