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 | NCT04571970 (ClinicalTrials.gov) | March 11, 2021 | 23/9/2020 | RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) | A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome) | Mucopolysaccharidosis Type II (MPS II) | Genetic: RGX-121 | REGENXBIO Inc. | NULL | Active, not recruiting | 5 Years | 17 Years | Male | 6 | Phase 1/Phase 2 | United States;Canada |
| 2 | NCT03566043 (ClinicalTrials.gov) | September 27, 2018 | 1/5/2018 | CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) | A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome) | Mucopolysaccharidosis Type II (MPS II) | Genetic: RGX-121 | REGENXBIO Inc. | NULL | Recruiting | 4 Months | 5 Years | Male | 48 | Phase 2/Phase 3 | United States;Brazil |