19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


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

  
21 trials found
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
7EUCTR2021-005402-10-DE
(EUCTR)
23/03/202205/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;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
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
2Phase 3Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan
8EUCTR2021-002350-90-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
9EUCTR2021-002320-20-IT
(EUCTR)
14/01/202222/10/2021A 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]
Trade Name: Fabrazyme
Product Name: .
Product Code: [.]
INN or Proposed INN: AGALSIDASI BETA
Trade Name: Galafold
Product Name: .
Product Code: [.]
INN or Proposed INN: Migalastat
Trade Name: Replagal
Product Name: .
Product Code: [.]
INN or Proposed INN: AGALSIDASI ALFA
Product Name: Venglustat
Product Code: [SAR402671, GZ402671 o GZ/SAR402671]
INN or Proposed INN: venglustat malato
Other descriptive name: GZ/SAR402671
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNULLAuthorised-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;Germany;Netherlands;China;Japan;Korea, Republic of
10EUCTR2021-002350-90-IT
(EUCTR)
04/11/202126/01/2022A 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 - . Fabry 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 o GZ/SAR402671]
INN or Proposed INN: Venglustat malato
Other descriptive name: GZ/SAR402671
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNULLAuthorised-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
11EUCTR2021-002350-90-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
12EUCTR2014-002550-39-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
13EUCTR2014-004995-49-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
14EUCTR2014-004995-49-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
15NCT02489344
(ClinicalTrials.gov)
July 7, 201530/6/2015Evaluation 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 DiseaseFabry DiseaseDrug: GZ/SAR402671Genzyme, a Sanofi CompanyNULLCompleted18 YearsN/AMale8Phase 2United States;France;Poland;Russian Federation;United Kingdom
16EUCTR2014-004995-49-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
17NCT02228460
(ClinicalTrials.gov)
November 201427/8/2014Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 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 DiseaseFabry DiseaseDrug: GZ/SAR402671Genzyme, a Sanofi CompanyNULLCompleted18 Years49 YearsMale11Phase 2United States;France;Poland;Russian Federation;United Kingdom;Czech Republic;Czechia
18EUCTR2013-005324-41-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
19EUCTR2013-005324-41-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
20EUCTR2021-002320-20-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
21EUCTR2021-005402-10-FR
(EUCTR)
27/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;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
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLNAFemale: yes
Male: yes
50Phase 3United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan