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 | NCT04411654 (ClinicalTrials.gov) | June 29, 2021 | 11/5/2020 | Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants With Type 2 Gaucher Disease | Gaucher Disease, Type 2 | Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone | Prevail Therapeutics | Eli Lilly and Company | Recruiting | N/A | 24 Months | All | 15 | Phase 1/Phase 2 | United States;United Kingdom |