Rp-l401 (DrugBank: -)
1 disease| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 326 | Osteopetrosis | 1 |
| 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 | NCT04525352 (ClinicalTrials.gov) | October 2020 | 11/8/2020 | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis | A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene | Infantile Malignant Osteopetrosis | Biological: RP-L401 | Rocket Pharmaceuticals Inc. | NULL | Not yet recruiting | 1 Month | N/A | All | 2 | Phase 1 | NULL |