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 | NCT01372228 (ClinicalTrials.gov) | April 2011 | 10/6/2011 | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Hurler Syndrome (MPS I);Hurler-Scheie Syndrome;Hunter Syndrome (MPS II);Sanfilippo Syndrome (MPS III);Krabbe Disease (Globoid Leukodystrophy);Metachromatic Leukodystrophy (MLD);Adrenoleukodystrophy (ALD and AMN);Sandhoff Disease;Tay Sachs Disease;Pelizaeus Merzbacher (PMD);Niemann-Pick Disease;Alpha-mannosidosis | Biological: hematopoietic stem cell infusion | Talaris Therapeutics Inc. | Duke University | Active, not recruiting | N/A | N/A | All | 30 | Phase 1/Phase 2 | United States |