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 | NCT01299727 (ClinicalTrials.gov) | March 1, 2011 | 16/2/2011 | Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | Sanfilippo Syndrome | Biological: rhHNS-10 mg;Biological: rhHNS-45 mg;Biological: rhHNS-90 mg | Shire | NULL | Terminated | 3 Years | N/A | All | 12 | Phase 1/Phase 2 | Netherlands;United Kingdom |