Venglustat malate ( DrugBank: Venglustat )
3 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 6 | Parkinson disease | 4 |
| 19 | Lysosomal storage disease | 15 |
| 67 | Polycystic kidney disease | 4 |
6. Parkinson disease
Clinical trials : 2,307 / Drugs : 2,007 - (DrugBank : 349) / Drug target genes : 188 - Drug target pathways : 199
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2016-000657-12-AT (EUCTR) | 03/04/2018 | 17/10/2016 | A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation | Multicenter, 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 290 | Phase 2 | United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden | ||
| 2 | EUCTR2016-000657-12-DE (EUCTR) | 26/01/2017 | 17/10/2016 | A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation | Multicenter, 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 290 | Phase 2 | United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Australia;Germany;Norway;Japan;Sweden | ||
| 3 | EUCTR2016-000657-12-PT (EUCTR) | 09/01/2017 | 25/10/2016 | A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation | Multicenter, 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 290 | Phase 2 | Germany;Norway;Japan;Portugal;United States;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Sweden | ||
| 4 | EUCTR2016-000657-12-SE (EUCTR) | 09/11/2016 | 12/10/2016 | A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation | Multicenter, 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 290 | Phase 2 | United States;Portugal;Taiwan;Greece;Spain;Austria;Israel;United Kingdom;Italy;France;Canada;Singapore;Germany;Norway;Japan;Sweden |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-002320-20-NL (EUCTR) | 21/06/2022 | 16/03/2022 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A 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 développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of | ||
| 2 | EUCTR2021-002320-20-DK (EUCTR) | 05/05/2022 | 12/11/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A 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 développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Germany;Netherlands;Norway;China;Japan;Korea, Republic of | ||
| 3 | EUCTR2021-002350-90-AT (EUCTR) | 22/04/2022 | 28/10/2021 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease | A 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 & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China | ||
| 4 | EUCTR2021-002320-20-PL (EUCTR) | 12/04/2022 | 11/01/2022 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A 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 développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of | ||
| 5 | EUCTR2021-002320-20-NO (EUCTR) | 11/04/2022 | 12/10/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A 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 développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Netherlands;Norway;Germany;China;Japan;Korea, Republic of | ||
| 6 | EUCTR2021-002350-90-FI (EUCTR) | 01/04/2022 | 06/10/2021 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease | A 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 & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China | ||
| 7 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/2022 | 05/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A 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 & développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2 | Phase 3 | Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan | ||
| 8 | EUCTR2021-002350-90-PL (EUCTR) | 08/03/2022 | 28/10/2021 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease | A 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 & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | France;Mexico;Canada;Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China | ||
| 9 | EUCTR2021-002350-90-GR (EUCTR) | 01/11/2021 | 01/11/2021 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease | A 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 & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Poland;Brazil;Romania;Denmark;Bulgaria;Norway;Netherlands;Germany;China | ||
| 10 | EUCTR2020-003120-17-FR (EUCTR) | 18/05/2021 | 11/03/2021 | Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3 | A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT | Gaucher disease type 3 MedDRA version: 20.0;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: GZ402671 INN or Proposed INN: Venglustat malate Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat malate Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2;Phase 3 | United States;Taiwan;Turkey;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden | ||
| 11 | EUCTR2020-003120-17-DE (EUCTR) | 12/04/2021 | 23/02/2021 | Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3 | A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT | Gaucher disease type 3 MedDRA version: 20.0;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: GZ402671 INN or Proposed INN: Venglustat malate Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat malate Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2;Phase 3 | United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden | ||
| 12 | EUCTR2020-003120-17-HU (EUCTR) | 06/04/2021 | 24/02/2021 | Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3 | A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT | Gaucher disease type 3 MedDRA version: 20.0;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: GZ402671 INN or Proposed INN: Venglustat malate Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat malate Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2;Phase 3 | United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden | ||
| 13 | EUCTR2021-002320-20-ES (EUCTR) | 19/11/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A 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 développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of | |||
| 14 | EUCTR2021-005402-10-FR (EUCTR) | 27/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A 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 & développement | NULL | NA | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan | |||
| 15 | EUCTR2020-003120-17-SE (EUCTR) | 17/02/2021 | Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3 | A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT | Gaucher disease type 3 MedDRA version: 20.0;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: GZ402671 INN or Proposed INN: Venglustat malate Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat malate Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2;Phase 3 | United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden |
67. Polycystic kidney disease
Clinical trials : 221 / Drugs : 212 - (DrugBank : 55) / Drug target genes : 40 - Drug target pathways : 151
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2020-004400-34-NL (EUCTR) | 12/05/2021 | 04/02/2021 | Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustat | Multicenter, 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 & Développement | NULL | Not Recruiting | Female: yes Male: yes | 640 | Phase 3 | Portugal;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of | ||
| 2 | EUCTR2020-004400-34-BE (EUCTR) | 11/05/2021 | 18/02/2021 | Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustat | Multicenter, 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 & Développement | NULL | Not Recruiting | Female: yes Male: yes | 640 | Phase 3 | Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of | ||
| 3 | EUCTR2020-004400-34-PT (EUCTR) | 30/04/2021 | 05/03/2021 | Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustat | Multicenter, 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 & Développement | NULL | Not Recruiting | Female: yes Male: yes | 640 | Phase 3 | Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of | ||
| 4 | EUCTR2020-004400-34-DE (EUCTR) | 23/03/2021 | 25/02/2021 | Long-term treatment of autosomal dominant polycystic kidney disease (ADPKD) with venglustat | Multicenter, 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 & Développement | NULL | Not Recruiting | Female: yes Male: yes | 640 | Phase 3 | Portugal;Czechia;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Poland;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Korea, Republic of |