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 | NCT02084121 (ClinicalTrials.gov) | March 2014 | 7/3/2014 | Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use) | Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use) | Metachromatic Leukodystrophy | Biological: Enriched Hematopoetic Stem Cell Infusion | Talaris Therapeutics Inc. | Duke University | No longer available | 3 Years | N/A | Male | United States | ||
| 2 | NCT02021266 (ClinicalTrials.gov) | April 2013 | 19/12/2013 | Single Patient Expanded Access Protocol: Metabolic Boost | Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Metachromatic Leukodystrophy | Biological: Enriched Hematopoetic Stem Cell Infusion | Talaris Therapeutics Inc. | Duke University | No longer available | N/A | N/A | Female | Phase 1/2 | United States |