19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
Showing 1 to 10 of 28 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 | NCT05222906 (ClinicalTrials.gov) | March 30, 2022 | 20/1/2022 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 Study to Evaluate the Efficacy and Safety of Venglustatin Adult and Pediatric Patients With Gaucher ... | 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) A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Stu ... | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Not yet recruiting | 12 Years | N/A | All | 40 | Phase 3 | NULL |
| 2 | 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 Study to Evaluate the Effect of VenglustatTablets on Neuropathic and Abdominal Pain in Male and Fe ... | 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 A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Ven ... | Fabry Disease | Drug: Venglustat (GZ402671);Drug: Placebo | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | N/A | All | 114 | Phase 3 | United States |
| 3 | 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 study to evaluate the effect of venglustattablets on left ventricular mass index in male and femal ... | 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 - . A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | 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] Fabry's disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ... | 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 Trade Name: Fabrazyme Product Name: . Product Code: [.] INN or Proposed INN: AGALSIDASI BETA Trade Name: ... | 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 United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;D ... | ||
| 4 | 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 study to evaluate the effect of venglustattablets on neuropathic and abdominal pain in male and fe ... | 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 - . A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of ven ... | 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] Fabry Disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Syst ... | Product Name: Venglustat Product Code: [SAR402671, GZ402671 o GZ/SAR402671] INN or Proposed INN: Venglustat malato Other descriptive name: GZ/SAR402671 Product Name: Venglustat Product Code: [SAR402671, GZ402671 o GZ/SAR402671] INN or Proposed INN: Vengl ... | 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 Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;A ... | ||
| 5 | 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 study to evaluate the effect of venglustattablets on neuropathic and abdominal pain in male and fe ... | 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 A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of ven ... | 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] MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Clas ... | 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: Venglu ... | 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 Greece;Finland;Ukraine;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;A ... | ||
| 6 | 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 venglustatin combination with Cerezyme in adul ... | 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 | ||
| 7 | 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 ... | ||
| 8 | 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 venglustatin combination with Cerezyme in adul ... | 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 | ||
| 9 | 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 venglustatin combination with Cerezyme in adul ... | 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 | ||
| 10 | 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 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 A multicenter, multinational, randomized, double-blind, placebo-controlled study to assess the effic ... | Tay-Sachs Disease, Sandhoff Disease | Drug: venglustat GZ402671 - Pharmaceutical form: tablet - Route of administration: oral Drug: placebo - Pharmaceutical form: tablet - Route of administration: oral Drug: venglustatGZ402671 - Pharmaceutical form: tablet - Route of administration: oral Drug: placebo - P ... | 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 Spain;United States;Brazil;Russian Federation;United Kingdom;Argentina;Austria;Czechia;Germany;Italy ... |