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 24 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 Eliglustatwith or without Imiglucerase in Pediatric Patients with Gaucher Dis ... | 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 Eliglustatwith or without Imiglucerase in Pediatric Patients with Gaucher Dis ... | 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 Eliglustatwith or without Imiglucerase in Pediatric Patients with Gaucher Dis ... | 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 Eliglustatwith or without Imiglucerase in Pediatric Patients with Gaucher Dis ... | 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 Eliglustatwith or without Imiglucerase in Pediatric Patients with Gaucher Dis ... | 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 | NCT03519646 (ClinicalTrials.gov) | April 23, 2018 | 20/3/2018 | Eliglustat on Gaucher Disease Type IIIB | Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases. Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disea ... | Gaucher Disease, Type III | Drug: Eliglustat | National Taiwan University Hospital | Sanofi | Completed | 6 Years | N/A | All | 4 | N/A | Taiwan |
| 7 | 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 EliglustatT ... | 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 EliglustatTartrate Given 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 |
| 8 | 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 EliglustatTar ... | 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 EliglustatTartrate Given as a Sing ... | Gaucher Disease | Drug: eliglustat | Sanofi | NULL | Completed | 18 Years | 79 Years | All | 32 | Phase 1 | United States |
| 9 | NCT02422654 (ClinicalTrials.gov) | April 2015 | 14/4/2015 | Taste Evaluation of Different Liquid Formulations With Eliglustat | A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, Repeated-doses (With no Ingestion) Study to Assess the Palatability of Eliglustat Prototype Liquid Formulations in Healthy Subjects A Single-blind, Randomized, Unbalanced Crossover Design With 5 Vehicles, 5 Periods, and 5 Sequences, ... | Gaucher Disease | Drug: eliglustat | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 55 Years | Both | 8 | Phase 1 | United States |
| 10 | 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 EliglustatTartrate (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 ... |