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 | NCT03580083 (ClinicalTrials.gov) | April 3, 2019 | 18/6/2018 | RGX-111 Gene Therapy in Patients With MPS I | A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type I | Mucopolysaccharidosis Type I (MPS I);Hurler Syndrome;Hurler-Scheie Syndrome | Genetic: RGX-111 | REGENXBIO Inc. | NULL | Active, not recruiting | 4 Months | N/A | All | 8 | Phase 1/Phase 2 | United States;Brazil;Israel |