Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII ( DrugBank: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 1 |
19. ライソゾーム病
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 | NCT05665166 (ClinicalTrials.gov) | February 2023 | 7/12/2022 | Autologous Ex-vivo Gene Modified HSCT in MPSII | A Phase I/II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11B Lentiviral Vector Encoding Human IDS Tagged With ApoEII in Patients With Neuronopathic Mucopolysaccharidosis Type II (nMPS II, Hunters Syndrome) | Mucopolysaccharidosis II | Genetic: Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII | University of Manchester | Manchester University NHS Foundation Trust;AVROBIO;CTI Clinical Trial and Consulting Services;Great Ormond Street Hospital for Children NHS Foundation Trust | Not yet recruiting | 3 Months | 12 Months | Male | 5 | Phase 1/Phase 2 | NULL |