19. Lysosomal storage disease
784 clinical trials, 673 drugs (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04656600 (ClinicalTrials.gov) | December 31, 2020 | 4/12/2020 | Phase IV Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ? | A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type ? | Gaucher's Disease | Drug: Cerezyme® / Imiglucerase | Sanofi | NULL | Not yet recruiting | 2 Years | N/A | All | 12 | Phase 4 | NULL |
| 2 | 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 | 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 | 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] | 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 | |||
| 3 | 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 | 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 | 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] | 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 | |||
| 4 | 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 | 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 | 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 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 | ||
| 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 | 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 | 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 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 | ||
| 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 |
| 6 | NCT03485677 (ClinicalTrials.gov) | April 11, 2018 | 23/3/2018 | Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 | 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 | Gaucher's Disease Type I;Gaucher's Disease Type III | Drug: Eliglustat GZ385660;Drug: Imiglucerase GZ437843 | Sanofi | NULL | Recruiting | 2 Years | 17 Years | All | 60 | Phase 3 | Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom |
| 7 | NCT02843035 (ClinicalTrials.gov) | January 2017 | 20/7/2016 | 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 | Gaucher Disease Type 1-Gaucher Disease Type 3 | Drug: placebo;Drug: venglustat (GZ/SAR402671);Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 49 | Phase 2;Phase 3 | United States;Germany;Japan;United Kingdom |
| 8 | NCT01951989 (ClinicalTrials.gov) | November 2012 | 2/9/2013 | Intra-monocyte Imiglucerase Kinetics in Gaucher Disease | Study of Intra-monocytic Imiglucerase Kinetic and Its Correlation With Clinical and Biological Gaucher Disease | Gaucher Disease | Drug: Imiglucérase (drug) pharmacokinetics | University Hospital, Clermont-Ferrand | NULL | Recruiting | 12 Years | N/A | Both | 60 | Phase 2 | France |
| 9 | NCT02770625 (ClinicalTrials.gov) | September 2011 | 7/5/2016 | Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease | A Multicenter, Open-Label Phase III Study to Evaluate the Safety and Efficacy of ISU302 (Imiglucerase for Injection) in Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Drug: ISU302 | ISU Abxis Co., Ltd. | NULL | Completed | 2 Years | N/A | All | 8 | Phase 3 | NULL |
| 10 | NCT01161914 (ClinicalTrials.gov) | January 2011 | 11/7/2010 | The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease | A Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher Disease | Gaucher Disease | Drug: Cerezyme®;Drug: ISU302 | ISU Abxis Co., Ltd. | NULL | Withdrawn | 2 Years | 75 Years | Both | 0 | Phase 3 | NULL |
| 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 |
| 11 | NCT01136304 (ClinicalTrials.gov) | April 2010 | 31/5/2010 | Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1) | Retrospective and Prospective Validation of a Disease Severity Score System (DS3) for Adults With Type 1 Gaucher Disease (GD1) | Gaucher Disease | Drug: Imiglucerase | University Research Foundation for Lysosomal Storage Diseases, Inc. | University of Pittsburgh | Completed | 18 Years | N/A | Both | 173 | N/A | United States |
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | EUCTR2008-005825-12-GB (EUCTR) | 17/12/2009 | 28/04/2009 | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Gaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Spain;Germany;Italy;United Kingdom | |||
| 15 | EUCTR2008-005825-12-DE (EUCTR) | 10/12/2009 | 16/09/2009 | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Gaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: UPLYSO Product Code: Taliglucerase alfa INN or Proposed INN: Taliglucerase alfa Other descriptive name: Recombinant Human Glucocerebrosidase (prGCD) | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Spain;Germany;Italy;United Kingdom | ||
| 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 |
| 16 | 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 | ||
| 17 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | Czech Republic;Germany;United Kingdom;Netherlands;France;Spain;Italy | ||
| 18 | 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 | Germany;United Kingdom;Czech Republic;Netherlands;France;Spain;Italy | ||
| 19 | 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 |
| 20 | NCT00954460 (ClinicalTrials.gov) | August 2009 | 5/8/2009 | Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease | Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Drug: velaglucerase alfa | Shire | NULL | Approved for marketing | 3 Years | N/A | Both | N/A | United States | |
| 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 |
| 21 | EUCTR2008-005825-12-IT (EUCTR) | 28/07/2009 | 16/09/2009 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | Gaucher disease MedDRA version: 9.1;Level: SOC;Classification code 10029205 | Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD | PROTALIX BIOTHERAPEUTICS | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| 22 | EUCTR2008-005825-12-ES (EUCTR) | 04/06/2009 | 06/03/2009 | Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Enfermedad de GaucherGaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: recombinant human glucocerebrosidase | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| 23 | EUCTR2007-005516-61-IT (EUCTR) | 06/02/2009 | 23/03/2009 | Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND | Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND | GAUCHER disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Trade Name: CEREZYME INN or Proposed INN: Imiglucerase Trade Name: ZAVESCA INN or Proposed INN: Miglustat | ISTITUTO GIANNINA GASLINI | NULL | Not Recruiting | Female: yes Male: yes | Italy | ||||
| 24 | NCT00712348 (ClinicalTrials.gov) | December 2008 | 7/7/2008 | Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase | Gaucher Disease | Drug: Taliglucerase alfa | Pfizer | NULL | Completed | 2 Years | N/A | All | 31 | Phase 3 | United States;Australia;Canada;Israel;Spain;United Kingdom;Serbia |
| 25 | EUCTR2007-007164-20-ES (EUCTR) | 20/05/2008 | 21/02/2008 | Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 | Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 | Tratamiento de la enfermedad de Gaucher MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Trade Name: CEREZYME INN or Proposed INN: IMIGLUCERASA Trade Name: MIGLUSTAT (ZAVESCA) INN or Proposed INN: MIGLUSTAT | INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD | NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 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 |
| 26 | EUCTR2006-006304-11-IT (EUCTR) | 14/04/2008 | 04/08/2008 | A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND | A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND | Patients with type 1 Gaucher disease who previously received imiglucerase MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB | SHIRE HUMAN GENETIC THERAPIES, INC. | NULL | Not Recruiting | Female: yes Male: yes | 40 | United Kingdom;Spain;Italy | |||
| 27 | EUCTR2007-002840-21-IT (EUCTR) | 25/02/2008 | 15/04/2008 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039 | Patients with Gaucher disease of type 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human Glucocerebrosidase Product Code: GA-GCB Product Name: imiglucerasi INN or Proposed INN: Imiglucerase | SHIRE HUMAN GENETIC THERAPIES, INC. | NULL | Not Recruiting | Female: yes Male: yes | 32 | United Kingdom;Spain;Italy | |||
| 28 | NCT00553631 (ClinicalTrials.gov) | January 2008 | 1/11/2007 | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease | Gaucher Disease, Type 1 | Biological: velaglucerase alfa;Biological: imiglucerase | Shire | NULL | Completed | 2 Years | N/A | All | 34 | Phase 3 | United States;Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom |
| 29 | EUCTR2007-002840-21-GB (EUCTR) | 29/11/2007 | 11/10/2007 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039 | Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 32 | United Kingdom;Spain;Italy | |||
| 30 | EUCTR2007-002840-21-ES (EUCTR) | 30/10/2007 | 18/07/2007 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 | Type I Gaucher diseaseEnfermedad de Gaucher tipo 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme INN or Proposed INN: IMIGLUCERASE | Shire Human Genetic Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | United Kingdom;Spain;Italy | |||
| 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 |
| 31 | EUCTR2006-006304-11-ES (EUCTR) | 27/08/2007 | 04/03/2010 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034 | Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | United Kingdom;Spain;Italy | |||
| 32 | NCT00478647 (ClinicalTrials.gov) | July 25, 2007 | 23/5/2007 | Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase | A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase | Gaucher Disease | Biological: GA-GCB (velaglucerase alfa) | Shire | NULL | Completed | 2 Years | N/A | All | 40 | Phase 2;Phase 3 | United States;Israel;Poland;Spain;United Kingdom |
| 33 | EUCTR2006-006304-11-GB (EUCTR) | 03/07/2007 | 12/02/2007 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034 | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2;Phase 3 | Spain;Germany;Italy;United Kingdom | |||
| 34 | NCT00365131 (ClinicalTrials.gov) | December 1997 | 15/8/2006 | A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. | Gaucher Disease Type I;Cerebroside Lipidosis Syndrome;Clucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic Form | Drug: Cerezyme (imiglucerase for injection) | Genzyme, a Sanofi Company | NULL | Completed | 10 Years | 65 Years | Both | 40 | Phase 4 | United States | |
| 35 | EUCTR2006-006304-11-DE (EUCTR) | 02/11/2007 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034 | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2;Phase 3 | Spain;Germany;Italy;United Kingdom | |||
| 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 |
| 36 | EUCTR2016-000301-37-IT (EUCTR) | 20/06/2018 | Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3 | 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 | 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 Corporation | NULL | NA | Female: yes Male: yes | 120 | Phase 3 | France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden | |||
| 37 | EUCTR2008-005825-12-Outside-EU/EEA (EUCTR) | 10/04/2012 | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | Gaucher disease 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: Taliglucerase alfa - Recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: TALIGLUCERASE ALFA | Protalix Biotherapeutics | NULL | NA | Female: yes Male: yes | 30 | Canada;Israel;United Kingdom;United States |