19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 | NCT03897361 (ClinicalTrials.gov) | July 8, 2019 | 6/2/2019 | Stem Cell Gene Therapy for Cystinosis | A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector | Lysosomal Storage Diseases;Cystinosis | Genetic: CTNS-RD-04 or CTNS-RD-04-LB (where the suffix -LB stands for LentiBOOST) | University of California, San Diego | California Institute for Regenerative Medicine (CIRM);Cystinosis Research Foundation | Recruiting | 18 Years | N/A | All | 6 | Phase 1/Phase 2 | United States |