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

  
6 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1EUCTR2016-000301-37-NL
(EUCTR)
06/03/201928/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open 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 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]
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
Genzyme CorporationNULLAuthorised-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): noFrance;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
2EUCTR2016-000301-37-GB
(EUCTR)
09/01/201917/06/2019Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open 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 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]
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
Genzyme CorporationNULLAuthorised-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): noFrance;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
3EUCTR2016-000301-37-SE
(EUCTR)
09/10/201805/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open 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 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]
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
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3Taiwan;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Canada;Argentina;Netherlands;Japan;Sweden
4EUCTR2016-000301-37-IT
(EUCTR)
02/07/201820/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open 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 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]
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
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden
5EUCTR2016-000301-37-ES
(EUCTR)
21/06/201806/07/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open 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 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]
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
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
6NCT03519646
(ClinicalTrials.gov)
April 23, 201820/3/2018Eliglustat on Gaucher Disease Type IIIBEvaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.Gaucher Disease, Type IIIDrug: EliglustatNational Taiwan University HospitalSanofiCompleted6 YearsN/AAll4N/ATaiwan