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 | 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 | ||
| 2 | EUCTR2012-003427-38-Outside-EU/EEA (EUCTR) | 07/04/2016 | Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease | A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease | Type 3 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] - Nervous System Diseases [C10] | Trade Name: VPRIV Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: VELAGLUCERASE ALFA | Shire Human Genetic Therapies | NULL | NA | Female: yes Male: yes | 6 | Tunisia;Egypt;India |