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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-001578-17-DE (EUCTR) | 12/09/2016 | 22/12/2015 | An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease | An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease | Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 4 | United States;Spain;Turkey;Israel;Germany;Italy;United Kingdom;India | ||
| 2 | EUCTR2015-001578-17-ES (EUCTR) | 25/02/2016 | 13/01/2016 | An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease | An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease | Gaucher Disease MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 4 | United States;Canada;Spain;Israel;Germany;Italy;United Kingdom | ||
| 3 | EUCTR2015-001578-17-GB (EUCTR) | 17/02/2016 | 21/12/2015 | An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease | An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect ofTreatment with Velaglucerase alfa on Bone-related Pathology inTreatment-naïve Patients with Type 1 Gaucher Disease | Gaucher Disease MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: VPRIV 400 Units powder for solution of infusion INN or Proposed INN: VELAGLUCERASE ALFA Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 19 | Phase 4 | United States;Canada;Spain;Israel;Germany;United Kingdom | ||
| 4 | EUCTR2008-001965-27-ES (EUCTR) | 23/12/2008 | 17/09/2008 | Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Enfermedad de Gaucher de tipo IType 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 | 102 | Spain;United Kingdom | |||
| 5 | EUCTR2008-001965-27-GB (EUCTR) | 29/10/2008 | 15/07/2008 | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa Product Name: Gene-Activated Human Glucocerebrosidase 200U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;United Kingdom | ||
| 6 | 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 | |||
| 7 | 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 | |||
| 8 | 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 | |||
| 9 | 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 | |||
| 10 | 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 | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Spain;Germany;Italy;United Kingdom | ||
| 11 | 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 |