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 | NCT00975689 (ClinicalTrials.gov) | August 2009 | 10/9/2009 | Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine | Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine | Niemann-Pick Disease, Type C | Drug: N-Acetyl Cysteine | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | University of Oxford;Washington University School of Medicine;National Human Genome Research Institute (NHGRI) | Completed | 12 Months | N/A | All | 35 | Phase 1/Phase 2 | United States |