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 | NCT02232477 (ClinicalTrials.gov) | May 2014 | 3/9/2014 | Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I | An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I | Mucopolysaccharidosis I;Cognitive Decline | Drug: Intrathecal recombinant human alpha iduronidase | agnes chen | University of Minnesota;UCSF Benioff Children's Hospital Oakland;University of California, Los Angeles;The Ryan Foundation;Rare Diseases Clinical Research Network;National Institute of Neurological Disorders and Stroke (NINDS) | Terminated | 6 Years | N/A | All | 6 | N/A | United States |