19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
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 |
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1 | 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 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 | 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] | 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 | 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 | ||
2 | EUCTR2009-015811-42-GR (EUCTR) | 25/01/2011 | 19/05/2011 | 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, 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 | Gaucher Disease Type I MedDRA version: 12.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | 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 | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 234 | Phase 3 | Portugal;Greece;Austria;Sweden | ||
3 | 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 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 | 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] | 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 | 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 | ||
4 | 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 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 | 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] | 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 | 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 | ||
5 | NCT01074944 (ClinicalTrials.gov) | June 2010 | 23/2/2010 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) | 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 | Gaucher Disease | Drug: Eliglustat tartrate | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | All | 170 | Phase 3 | United States;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden |
6 | EUCTR2008-005223-28-ES (EUCTR) | 03/03/2010 | 17/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with CerezymeEstudio en fase 3, aleatorizado, multicéntrico, multinacional, abierto y con comparador activo para evaluar la eficacia y seguridad de Genz 112638 en pacientes con enfermedad de Gaucher de tipo 1 que se han estabilizado con Cerezyme - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with CerezymeEstudio en fase 3, aleatorizado, multicéntrico, multinacional, abierto y con comparador activo para evaluar la eficacia y seguridad de Genz 112638 en pacientes con enfermedad de Gaucher de tipo 1 que se han estabilizado con Cerezyme - ENCORE | Enfermedad de Gaucher tipo IGaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: INN Not available Other descriptive name: N/A | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom | ||
7 | EUCTR2008-005222-37-BG (EUCTR) | 10/02/2010 | 11/11/2009 | A study with Eliglustat Tartrate (Genz-112638) in Patients With GaucherDisease | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE | Gaucher Disease type I MedDRA version: 16.1;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] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 36 | Phase 3 | United States;Serbia;Saudi Arabia;Lebanon;Israel;Russian Federation;Colombia;United Kingdom;India;Mexico;Canada;Jordan;Bulgaria;Netherlands;Tunisia | ||
8 | EUCTR2008-005223-28-IT (EUCTR) | 29/01/2010 | 03/12/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | Gaucher Disease Type 1 MedDRA version: 12.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 Trade Name: CEREZYME INN or Proposed INN: Imiglucerase | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;United Kingdom;Italy | ||
9 | EUCTR2008-005223-28-DE (EUCTR) | 21/01/2010 | 11/08/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have reached therapeutic goals with enzyme replacement therapy - ENCORE | Gaucher Disease type I MedDRA version: 18.0;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] | 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 Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
10 | EUCTR2008-005223-28-GB (EUCTR) | 16/11/2009 | 06/07/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | 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] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
11 | EUCTR2008-005222-37-GB (EUCTR) | 06/11/2009 | 18/06/2009 | A study with Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE | 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] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: eliglustat Other descriptive name: not applicable Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: eliglustat Other descriptive name: not applicable Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: eliglustat Other descriptive name: not applicable | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 36 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Serbia;United States;Saudi Arabia;Lebanon;Russian Federation;Israel;Colombia;United Kingdom;India;Mexico;Canada;Jordan;Bulgaria;Netherlands;Tunisia | ||
12 | EUCTR2008-005223-28-NL (EUCTR) | 04/11/2009 | 01/07/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 186 | Phase 3 | Czech Republic;Germany;United Kingdom;Netherlands;France;Spain;Italy | ||
13 | EUCTR2008-005222-37-NL (EUCTR) | 04/11/2009 | 10/06/2009 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE | Gaucher Disease type I MedDRA version: 13.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders | 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 | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 3 | Bulgaria;Netherlands;United Kingdom | ||
14 | NCT00891202 (ClinicalTrials.gov) | November 2009 | 30/4/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE) | Gaucher Disease, Type 1 | Drug: Eliglustat tartrate;Drug: Placebo | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | All | 40 | Phase 3 | United States;Bulgaria;Canada;Colombia;India;Israel;Lebanon;Mexico;Russian Federation;Serbia;Tunisia;United Kingdom;Chile;Georgia;Jordan;Netherlands;Saudi Arabia |
15 | EUCTR2008-005223-28-FR (EUCTR) | 22/10/2009 | 05/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom | ||
16 | EUCTR2008-005223-28-CZ (EUCTR) | 23/09/2009 | 21/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat tartrate Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: recombinant human derived macrophage-targeted ß-glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | Italy;Spain;France;Netherlands;Czech Republic;United Kingdom;Germany | ||
17 | NCT00943111 (ClinicalTrials.gov) | September 2009 | 20/7/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) | Gaucher Disease, Type 1 | Drug: Eliglustat tartrate;Drug: Imiglucerase | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | All | 160 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;Egypt;France;Germany;Italy;Russian Federation;Spain;United Kingdom;Czech Republic;Netherlands;Turkey |
18 | EUCTR2005-004732-42-IT (EUCTR) | 12/10/2006 | 10/07/2007 | A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients - ND | A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients - ND | patients with diagnosis of Gaucher Type I disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Italy | ||
19 | NCT00358150 (ClinicalTrials.gov) | June 2006 | 27/7/2006 | A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients | A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients | Gaucher Disease, Type 1;Cerebroside Lipidosis Syndrome;Glucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic Form | Drug: Eliglustat tartrate | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 65 Years | All | 26 | Phase 2 | United States;Argentina;Israel;Italy;Mexico;Russian Federation |