Venglustat ( DrugBank: Venglustat )
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 | 5 |
| 19 | ライソゾーム病 | 32 |
| 67 | 多発性嚢胞腎 | 21 |
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. | 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 | NCT02906020 (ClinicalTrials.gov) | December 15, 2016 | 14/9/2016 | A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (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. | Parkinson's Disease | Drug: venglustat GZ/SAR402671;Drug: Placebo | Genzyme, a Sanofi Company | NULL | Terminated | 18 Years | 80 Years | All | 270 | Phase 2 | United States;Austria;Canada;France;Germany;Greece;Israel;Italy;Japan;Norway;Portugal;Singapore;Spain;Sweden;Taiwan;United Kingdom |
| 5 | 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. 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. | 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 | JPRN-jRCT2071220022 | 14/07/2022 | 25/06/2022 | 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 | Fabry's disease | Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Agalsidase alfa Pharmaceutical form: Concentrate for solution for infusion, Route of administration: IV infusion Drug: Agalsidase beta (GZ419828) Pharmaceutical form: Powder for concentrate for solution for infusion, Route of administration: IV infusion Drug: Migalastat Pharmaceutical form: Hard capsules, Route of administration: Oral | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | <= 65age old | Both | 90 | Phase 3 | China;Italy;Republic of Korea;Japan |
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | NCT05280548 (ClinicalTrials.gov) | May 3, 2022 | 4/3/2022 | 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 | Fabry's Disease | Drug: Venglustat (GZ402671);Drug: Agalsidase alfa;Drug: Agalsidase beta (GZ419828);Drug: Migalastat | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | 65 Years | All | 90 | Phase 3 | China;Czechia;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Spain;Taiwan;Turkey |
| 5 | 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 | ||
| 6 | NCT05222906 (ClinicalTrials.gov) | April 18, 2022 | 20/1/2022 | 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) | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 40 | Phase 3 | United States;Argentina;Canada;China;France;Germany;Hungary;Japan |
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | NCT05206773 (ClinicalTrials.gov) | March 11, 2022 | 20/10/2021 | 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 | Fabry Disease | Drug: Venglustat (GZ402671);Drug: Placebo | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | N/A | All | 114 | Phase 3 | United States;Argentina;Brazil;Canada;China;Finland;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;United Kingdom |
| 12 | 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 | ||
| 13 | EUCTR2021-002320-20-IT (EUCTR) | 14/01/2022 | 22/10/2021 | 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] | 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 DEVELOPPEMENT | 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;Germany;Netherlands;China;Japan;Korea, Republic of | ||
| 14 | EUCTR2021-002350-90-IT (EUCTR) | 04/11/2021 | 26/01/2022 | 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 - . | 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 DEVELOPPEMENT | 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 | ||
| 15 | 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 | ||
| 16 | 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 | ||
| 17 | EUCTR2019-002375-34-AT (EUCTR) | 27/04/2021 | 06/12/2019 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850 MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 104 | Phase 3 | United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 18 | 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 | ||
| 19 | 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 | ||
| 20 | JPRN-jRCT2021200023 | 27/10/2020 | 09/10/2020 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late onset GM2 gangliosidosis and ultra rare diseases within the same and similar glucosylceramide-based sphingolipid pathway | Tay-Sachs Disease, Sandhoff Disease | Drug: venglustat GZ402671 - Pharmaceutical form: tablet - Route of administration: oral Drug: placebo - Pharmaceutical form: tablet - Route of administration: oral | Tanaka Tomoyuki | NULL | Not Recruiting | >= 2age old | Not applicable | Both | 77 | Phase 3 | Spain;United States;Brazil;Russian Federation;United Kingdom;Argentina;Austria;Czechia;Germany;Italy;Portugal;Turkey;France;Japan |
| 21 | EUCTR2019-002375-34-DE (EUCTR) | 23/07/2020 | 25/11/2019 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850 MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 104 | Phase 3 | United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 22 | NCT04221451 (ClinicalTrials.gov) | June 29, 2020 | 6/1/2020 | A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 | A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway | Tay-Sachs Disease Sandhoff Disease | Drug: venglustat GZ402671;Drug: placebo | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 2 Years | N/A | All | 74 | Phase 3 | United States;Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom |
| 23 | EUCTR2019-002375-34-CZ (EUCTR) | 26/05/2020 | 26/05/2020 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850 MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 83 | Phase 3 | United States;Portugal;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 24 | EUCTR2019-002375-34-PT (EUCTR) | 25/05/2020 | 03/12/2019 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850 MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 104 | Phase 3 | Portugal;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 25 | JPRN-jRCT2021220001 | 20/04/2020 | 13/04/2022 | 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-naive or untreated for at least 6 months | Fabry Disease | Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Placebo Pharmaceutical form: Tablet, Route of administration: Oral | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | Not applicable | Both | 114 | Phase 3 | United States;Japan |
| 26 | EUCTR2019-002375-34-GB (EUCTR) | 23/03/2020 | 27/11/2019 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 100000004850 MedDRA version: 23.0;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 83 | Phase 3 | United States;Portugal;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 27 | EUCTR2019-002375-34-IT (EUCTR) | 04/03/2020 | 17/06/2021 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety ofvenglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: [GZ402671] INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: [GZ402671] INN or Proposed INN: venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: [GZ402671] INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | GENZYME CORPORATION | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 83 | Phase 3 | United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Argentina;Brazil;Germany;Japan | ||
| 28 | NCT02843035 (ClinicalTrials.gov) | January 4, 2017 | 20/7/2016 | Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | Gaucher Disease Type 1;Gaucher Disease Type 3 | Drug: venglustat (GZ402671);Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 18 Years | N/A | All | 13 | Phase 2 | United States;Germany;Japan;United Kingdom |
| 29 | 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 | |||
| 30 | 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 | |||
| 31 | 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 | |||
| 32 | EUCTR2019-002375-34-FR (EUCTR) | 19/12/2019 | A multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, and safety of venglustat in late-onset GM2 | A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmacodynamics, pharmacokinetics, safety, and tolerability of venglustat in late-onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) together with a separate basket for juvenile/adolescent late-onset GM2 gangliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway - AMETHIST | Tay-Sachs disease Sandhoff disease MedDRA version: 20.0;Level: PT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.1;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | Genzyme Corporation | NULL | NA | Female: yes Male: yes | 83 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Czech Republic;Argentina;Brazil;Germany;Japan;Portugal;United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy |
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. | 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-IT (EUCTR) | 14/04/2021 | 08/06/2021 | 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 o GZ/SAR402671] INN or Proposed INN: venglustat malato Other descriptive name: GZ/SAR402671 | SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT | 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 | ||
| 5 | 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 | ||
| 6 | JPRN-jRCT2031200348 | 09/02/2021 | 05/02/2021 | 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 | Drug: Venglustat (GZ402671) Pharmaceutical form: capsule, Route of administration: oral | Tanaka Tomoyuki | NULL | Suspended | >= 18age old | Not applicable | Both | 640 | Phase 3 | United States;Japan |
| 7 | NCT04705051 (ClinicalTrials.gov) | February 9, 2021 | 8/1/2021 | 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) | Congenital Cystic Kidney Disease | Drug: Venglustat GZ402671 | Sanofi | NULL | Terminated | 18 Years | N/A | All | 24 | Phase 3 | United States;Australia;Belgium;Germany;Japan;Korea, Republic of;Netherlands;Spain;France;Romania |
| 8 | EUCTR2017-004084-12-AT (EUCTR) | 11/04/2019 | 14/01/2019 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | United 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 | ||
| 9 | EUCTR2017-004084-12-RO (EUCTR) | 25/03/2019 | 23/05/2022 | Study 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 836 | Phase 2 | Portugal;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 | ||
| 10 | EUCTR2017-004084-12-NL (EUCTR) | 13/02/2019 | 06/06/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | United 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 | ||
| 11 | JPRN-JapicCTI-184192 | 31/1/2019 | 05/11/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients. | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Polycystic kidney, autosomal dominant | Intervention name : GZ402671 INN of the intervention : venglustat Dosage And administration of the intervention : once daily for 24 months Control intervention name : Placebo INN of the control intervention : - Dosage And administration of the control intervention : once daily for 24 months | Sanofi K.K. | NULL | other | 18 | 55 | BOTH | 640 | Phase 2-3 | Japan, Asia except Japan, North America, South America, Europe, Oceania |
| 12 | EUCTR2017-004084-12-GB (EUCTR) | 28/01/2019 | 24/05/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 982 | Phase 2 | United 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 | ||
| 13 | EUCTR2017-004084-12-DK (EUCTR) | 07/11/2018 | 23/05/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | Portugal;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 | ||
| 14 | NCT03687554 (ClinicalTrials.gov) | October 5, 2018 | 26/9/2018 | Effect of Venglustat in Patients With Renal Impairment | A Phase I, Single-Center, Open-label, Single Dose Pharmacokinetic and Tolerability Study of GZ402671 in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function | Healthy Volunteers;Polycystic Kidney, Autosomal Dominant | Drug: Venglustat GZ/SAR402671 | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 79 Years | All | 24 | Phase 1 | United States |
| 15 | NCT03523728 (ClinicalTrials.gov) | October 4, 2018 | 1/5/2018 | A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Polycystic Kidney, Autosomal Dominant | Drug: Venglustat;Drug: Placebo | Genzyme, a Sanofi Company | NULL | Terminated | 18 Years | 55 Years | All | 478 | Phase 2/Phase 3 | United States;Argentina;Australia;Austria;Belgium;Canada;China;Czechia;Denmark;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Poland;Portugal;Romania;Spain;Taiwan;Turkey;United Kingdom |
| 16 | EUCTR2017-004084-12-BE (EUCTR) | 31/08/2018 | 31/05/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Germany;Netherlands;China;Japan;Korea, Republic of | ||
| 17 | EUCTR2017-004084-12-PT (EUCTR) | 06/08/2018 | 11/06/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | Portugal;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 | ||
| 18 | EUCTR2017-004084-12-IT (EUCTR) | 12/07/2018 | 17/06/2021 | Study 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: GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU Product Name: Venglustat Product Code: GZ/SAR402671 INN or Proposed INN: Venglustat Other descriptive name: Genz-682452-AU | GENZYME CORPORATION | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | Portugal;United States;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of | ||
| 19 | EUCTR2017-004084-12-DE (EUCTR) | 10/07/2018 | 22/05/2018 | A Medical Research Study Designed to Determine if Venglustat can be aFuture Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | United 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 | ||
| 20 | EUCTR2017-004084-12-CZ (EUCTR) | 03/07/2018 | 13/06/2018 | A Medical Research Study Designed to Determine if Venglustat can be a Future Treatment for ADPKD Patients | Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) - 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 982 | Phase 2 | United States;Portugal;Czechia;Taiwan;Spain;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;China;Japan;Korea, Republic of | ||
| 21 | EUCTR2017-004084-12-ES (EUCTR) | 05/06/2018 | 06/06/2018 | Study 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 Corporation | NULL | Authorised-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): no | Portugal;United States;Taiwan;Spain;Austria;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Romania;Australia;Denmark;Netherlands;Germany;Japan;China;Korea, Republic of |