SAR402671, GZ402671 or GZ/SAR402671 ( DrugBank: - )


3 diseases
告示番号疾患名(ページ内リンク)臨床試験数
6パーキンソン病7
19ライソゾーム病13
67多発性嚢胞腎14

6. パーキンソン病


臨床試験数 : 2,307 薬物数 : 2,007 - (DrugBank : 349) / 標的遺伝子数 : 188 - 標的パスウェイ数 : 199
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-000657-12-GR
(EUCTR)
04/04/201911/01/2019A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: -
Other descriptive name: Genz-682452-AU
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: -
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
290Phase 2Taiwan;United States;Portugal;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Germany;Norway;Japan;Sweden
2EUCTR2016-000657-12-AT
(EUCTR)
03/04/201817/10/2016A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
290Phase 2United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden
3EUCTR2016-000657-12-IT
(EUCTR)
16/02/201708/02/2017A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 19.1;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: 000000
Other descriptive name: Genz-682452-AU
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: 00000
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
276Phase 2United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden
4EUCTR2016-000657-12-DE
(EUCTR)
26/01/201717/10/2016A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
290Phase 2United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden
5EUCTR2016-000657-12-PT
(EUCTR)
09/01/201725/10/2016A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
290Phase 2Germany;Norway;Japan;Portugal;United States;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Sweden
6EUCTR2016-000657-12-SE
(EUCTR)
09/11/201612/10/2016A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
290Phase 2United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Germany;Norway;Japan;Sweden
7EUCTR2016-000657-12-ES
(EUCTR)
20/10/201624/10/2016A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene MutationMulticenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients with Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant - MOVES-PD Parkinson's disease (PD) carrying a GBA mutation
MedDRA version: 19.0;Level: PT;Classification code 10061536;Term: Parkinson's disease;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: NA
Other descriptive name: Genz-682452-AU
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: NA
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
276Phase 2United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-002320-20-NL
(EUCTR)
21/06/202216/03/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Galafold
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
2EUCTR2021-002320-20-DK
(EUCTR)
05/05/202212/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Germany;Netherlands;Norway;China;Japan;Korea, Republic of
3EUCTR2021-002350-90-AT
(EUCTR)
22/04/202228/10/2021A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry diseaseA randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China
4EUCTR2021-002320-20-PL
(EUCTR)
12/04/202211/01/2022A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT Fabry’s disease
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of
5EUCTR2021-002320-20-NO
(EUCTR)
11/04/202212/10/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s disease
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Netherlands;Norway;Germany;China;Japan;Korea, Republic of
6EUCTR2021-002350-90-FI
(EUCTR)
01/04/202206/10/2021A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry diseaseA randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months - PERIDOT
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China
7EUCTR2021-002350-90-PL
(EUCTR)
08/03/202228/10/2021A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry diseaseA randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months - PERIDOT
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3France;Mexico;Canada;Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China
8EUCTR2021-002350-90-GR
(EUCTR)
01/11/202101/11/2021A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry diseaseA randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months - PERIDOT
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China
9EUCTR2014-002550-39-DE
(EUCTR)
20/10/201625/04/2016GZ/SAR402671 in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3A 208-week three-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in combination with Cerezyme in adult patients with Gaucher disease type 3 - LEAP Gaucher disease
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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 CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 2United States;Japan;United Kingdom;Germany
10EUCTR2014-004995-49-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
11EUCTR2014-004995-49-PL
(EUCTR)
28/07/201515/06/2015Evaluation 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 Recruiting Female: no
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Czech Republic;Poland;Russian Federation;United Kingdom
12EUCTR2014-004995-49-FR
(EUCTR)
11/06/201525/06/2015Evaluation 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: 20.0;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 Recruiting Female: no
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Czech Republic;Poland;Russian Federation;United Kingdom
13EUCTR2021-002320-20-ES
(EUCTR)
19/11/2021A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry diseaseA randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy Fabry’s disease
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Replagal
INN or Proposed INN: Agalsidase alfa
Trade Name: Fabrazyme
INN or Proposed INN: Agalsidase beta
Trade Name: Galafold
INN or Proposed INN: Migalastat
Other descriptive name: Migalastat
sanofi-aventis recherche et développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of

67. 多発性嚢胞腎


臨床試験数 : 221 薬物数 : 212 - (DrugBank : 55) / 標的遺伝子数 : 40 - 標的パスウェイ数 : 151
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-004400-34-NL
(EUCTR)
12/05/202104/02/2021Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustatMulticenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD) - STAGED-PKD-EXT Congenital cystic kidney disease
MedDRA version: 20.0;Level: PT;Classification code 10010428;Term: Congenital cystic kidney disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
640Phase 3Portugal;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of
2EUCTR2020-004400-34-BE
(EUCTR)
11/05/202118/02/2021Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustatMulticenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD) - STAGED-PKD-EXT Congenital cystic kidney disease
MedDRA version: 20.0;Level: PT;Classification code 10010428;Term: Congenital cystic kidney disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
640Phase 3Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of
3EUCTR2020-004400-34-PT
(EUCTR)
30/04/202105/03/2021Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustatMulticenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD) - STAGED-PKD-EXT Congenital cystic kidney disease
MedDRA version: 20.0;Level: PT;Classification code 10010428;Term: Congenital cystic kidney disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
640Phase 3Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of
4EUCTR2020-004400-34-DE
(EUCTR)
23/03/202125/02/2021Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustatMulticenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD) - STAGED-PKD-EXT Congenital cystic kidney disease
MedDRA version: 20.0;Level: PT;Classification code 10010428;Term: Congenital cystic kidney disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: SAR402671, GZ402671 or GZ/SAR402671
INN or Proposed INN: venglustat malate
Other descriptive name: GZ/SAR402671
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
640Phase 3Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Korea, Republic of
5EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: 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
6EUCTR2017-004084-12-RO
(EUCTR)
25/03/201923/05/2022Study of Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)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) - SAVE-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 CorporationNULLNot RecruitingFemale: yes
Male: yes
836Phase 2Portugal;United States;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;Switzerland;France;Czech Republic;Canada;Belgium;Romania;Denmark;Australia;Germany;Netherlands;China;Japan;Korea, Republic of
7EUCTR2017-004084-12-NL
(EUCTR)
13/02/201906/06/2018A 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 CorporationNULLNot RecruitingFemale: yes
Male: yes
982Phase 2United States;Portugal;Czechia;Taiwan;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Argentina;Belgium;Romania;Australia;Denmark;Netherlands;Germany;Japan;China;Korea, Republic of
8EUCTR2017-004084-12-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
9EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: yes
Male: yes
982Phase 2Portugal;United States;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Denmark;Australia;Germany;Netherlands;China;Japan;Korea, Republic of
10EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: 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
11EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: yes
Male: yes
982Phase 2Portugal;United States;Czechia;Taiwan;Spain;Austria;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of
12EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: 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
13EUCTR2017-004084-12-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 CorporationNULLNot RecruitingFemale: 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
14EUCTR2017-004084-12-ES
(EUCTR)
05/06/201806/06/2018Study of Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)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) - SAVE-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 finished Female: yes
Male: yes
836 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noPortugal;United States;Taiwan;Spain;Austria;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;Japan;China;Korea, Republic of