326. 大理石骨病 Osteopetrosis Clinical trials / Disease details
臨床試験数 : 20 / 薬物数 : 51 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 81
| 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) | February 1, 2021 | 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. | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 1 Month | N/A | All | 2 | Phase 1 | United States |