Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the a-L-iduronidase cDNA, in their final formulation medium. ( 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 | NCT03488394 (ClinicalTrials.gov) | May 11, 2018 | 22/3/2018 | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant | Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human a-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant | Mucopolysaccharidosis IH | Genetic: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the a-L-iduronidase cDNA, in their final formulation medium. | IRCCS San Raffaele | Fondazione Telethon | Active, not recruiting | N/A | 11 Years | All | 8 | Phase 1/Phase 2 | Italy |