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

  
2 trials found
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1NCT05682144
(ClinicalTrials.gov)
January 15, 202312/12/2022ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS IA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and ScheieMucopolysaccharidosis IH/S;Mucopolysaccharidosis ISBiological: Autologous Plasmablasts (B cells)Immusoft CorporationNULLNot yet recruiting18 YearsN/AAll2Phase 1United States
2NCT04284254
(ClinicalTrials.gov)
December 202211/11/2019MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCTSleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic TransplantationMucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome);Mucopolysaccharidosis Type IH;MPS IH, Hurler SyndromeDrug: Autologous PlasmablastsMasonic Cancer Center, University of MinnesotaNULLWithdrawn3 Years8 YearsAll0Phase 1/Phase 2NULL