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 | NCT01238315 (ClinicalTrials.gov) | November 2010 | 5/11/2010 | Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis | A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis | Neuronal Ceroid Lipofuscinosis | Biological: HuCNS-SC | StemCells, Inc. | NULL | Withdrawn | 6 Months | 6 Years | Both | 0 | Phase 1 | United States |
| 2 | NCT00337636 (ClinicalTrials.gov) | May 2006 | 13/6/2006 | Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL) | A Phase I Study of the Safety and Preliminary Effectiveness of Human CNS Stem Cells (HuCNS-SC) in Patients With Neuronal Ceroid Lipofuscinosis Caused by Palmitoyl Protein Thioesterase 1 (PPT1) or Tripeptidyl Peptidase 1 (TPP-I) Deficiency | Neuronal Ceroid Lipofuscinosis | Procedure: Surgery to implant human CNS stem cells (HuCNS-SC);Drug: Medication to suppress the immune system | StemCells, Inc. | NULL | Completed | 18 Months | 12 Years | Both | 6 | Phase 1 | United States |