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 19 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 | EUCTR2016-000301-37-NL (EUCTR) | 06/03/2019 | 28/06/2018 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Di ... | Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokineti ... | Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;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] Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 1007 ... | Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerezyme Product Name: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Cerezyme Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Oth ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Swede ... | ||
| 2 | EUCTR2016-000301-37-GB (EUCTR) | 09/01/2019 | 17/06/2019 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Di ... | Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokineti ... | Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;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] Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 1007 ... | Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerezyme Product Name: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Cerezyme Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Oth ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Swede ... | ||
| 3 | EUCTR2016-000301-37-SE (EUCTR) | 09/10/2018 | 05/06/2018 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Di ... | Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokineti ... | Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;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] Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 1007 ... | Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerezyme Product Name: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Cerezyme Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 Other descriptive name: Eliglustat Tartrate – Powder for Oral Suspension Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Oth ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | Taiwan;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Canada;Argentina;Netherlands;Japan;Sweden Taiwan;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Canada;Argentina;Netherland ... | ||
| 4 | EUCTR2016-000301-37-IT (EUCTR) | 02/07/2018 | 20/06/2018 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Di ... | Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokineti ... | Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;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] Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 1007 ... | Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerezyme Product Name: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Cerezyme Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Oth ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Swede ... | ||
| 5 | EUCTR2016-000301-37-ES (EUCTR) | 21/06/2018 | 06/07/2018 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Di ... | Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokineti ... | Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;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] Congenital, hereditary and neonatal diseases MedDRA version: 20.0;Level: PT;Classification code 1007 ... | Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerezyme Product Name: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Cerezyme Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Other descriptive name: ELIGLUSTAT TARTRATE Trade Name: Cerdelga Product Name: Eliglustat Product Code: GZ385660 INN or Proposed INN: Eliglustat Oth ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Swede ... | ||
| 6 | NCT02536911 (ClinicalTrials.gov) | September 2015 | 28/8/2015 | A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat ... | An Open-label Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild and Moderate Hepatic Impairment, and in Matched Subjects With Normal Hepatic Function An Open-label Pharmacokinetic and Tolerability Study of Eliglustat TartrateGiven as a Single Dose in ... | Gaucher Disease | Drug: eliglustat | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 79 Years | All | 24 | Phase 1 | United States |
| 7 | NCT02536937 (ClinicalTrials.gov) | September 2015 | 28/8/2015 | A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Ta ... | An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat Tartrate Given as a Single Dose in Subjects With Mild, Moderate and Severe Renal Impairment, and in Matched Subjects With Normal Renal Function An Open-label Two-stage Pharmacokinetic and Tolerability Study of Eliglustat TartrateGiven as a Sing ... | Gaucher Disease | Drug: eliglustat | Sanofi | NULL | Completed | 18 Years | 79 Years | All | 32 | Phase 1 | United States |
| 8 | EUCTR2009-015811-42-PT (EUCTR) | 04/02/2011 | 09/11/2010 | A study with Eliglustat Tartrate (Genz-112638) in Patients with Gaucher disease to Evaluate Once Daily Dosing versus Twice Daily A study with Eliglustat Tartrate(Genz-112638) in Patients with Gaucher disease to Evaluate Once Dail ... | A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Sa ... | Gaucher Disease Type I MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Gaucher Disease Type I MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's d ... | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive n ... | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | United States;Serbia;Portugal;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;China;Japan;Sweden United States;Serbia;Portugal;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil ... | ||
| 9 | EUCTR2009-015811-42-AT (EUCTR) | 05/01/2011 | 22/11/2010 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) A Study of Eliglustat Tartrate(Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily ... | A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Sa ... | Gaucher Disease Type I MedDRA version: 15.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Gaucher Disease Type I MedDRA version: 15.0;Level: PT;Classification code 10018048;Term: Gaucher's d ... | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive n ... | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;Japan;China;Sweden Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil ... | ||
| 10 | EUCTR2009-015811-42-SE (EUCTR) | 20/12/2010 | 01/11/2010 | A study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease to Evaluate Once Daily versus Twice Daily Dosing A study of Eliglustat Tartrate(Genz-112638) in Patients with Gaucher Disease to Evaluate Once Daily ... | A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Sa ... | Gaucher Disease Type I MedDRA version: 16.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Gaucher Disease Type I MedDRA version: 16.0;Level: PT;Classification code 10018048;Term: Gaucher's d ... | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive n ... | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 170 | Phase 3 | Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;Japan;China;Sweden Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil ... |