19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | NCT00001234 (ClinicalTrials.gov) | January 1988 | 3/11/1999 | Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells | Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells | Gaucher's Disease | Genetic: human glucocerebrosidase cDNA | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 120 | Phase 1 | United States |