19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 15 diseases
| 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 | EUCTR2020-003120-17-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 A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adu ... | 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] Gaucher disease type 3 MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's d ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustatmalate Product Name: Ven ... | 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 | ||
| 2 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: ... | 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 United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;C ... | ||
| 3 | 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 A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adu ... | 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] Gaucher disease type 3 MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's d ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustatmalate Product Name: Ven ... | 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 | ||
| 4 | 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 A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adu ... | 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] Gaucher disease type 3 MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's d ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustatmalate Product Name: Ven ... | 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 | ||
| 5 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: ... | 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 United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;C ... | ||
| 6 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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: ... | 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 United States;Portugal;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Rep ... | ||
| 7 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: ... | 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 Portugal;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;C ... | ||
| 8 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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 Product Name: Venglustat Product Code: GZ402671 INN or Proposed INN: Venglustat Other descriptive name: ... | 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 United States;Portugal;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Rep ... | ||
| 9 | 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 multinational, randomized, double-blind, placebo-controlled study to assess the efficacy, pharmaco ... | 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | 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] Tay-Sachs diseaseSandhoff disease MedDRA version: 23.0;Level: LLT;Classification code 10043147;Term: ... | 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 Product Name: Venglustat Product Code: [GZ402671] INN or Proposed INN: Venglustat Other descriptive nam ... | 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 United States;Portugal;Czechia;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;C ... | ||
| 10 | EUCTR2014-002550-39-DE (EUCTR) | 20/10/2016 | 25/04/2016 | GZ/SAR402671 in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3 | A 208-week three-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in combination with Cerezyme in adult patients with Gaucher disease type 3 - LEAP A 208-week three-part, open-label, multicenter, multinational study of the safety, tolerability, pha ... | Gaucher disease MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Gaucher disease MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease ... | Product Code: SAR402671, GZ402671 or GZ/SAR402671 Other descriptive name: Genz-682452-AU Product Code: SAR402671, GZ402671 or GZ/SAR402671 Other descriptive name: Genz-682452-AU Product Code: SAR402671, GZ402671 or GZ/SAR402671 Other descriptive name: Genz-682452-AU Product Code: ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 2 | United States;Japan;United Kingdom;Germany |