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

  
1 trial found
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1NCT05487599
(ClinicalTrials.gov)
December 20, 20221/6/2022A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)Gaucher DiseaseBiological: LY3884961;Drug: Methylprednisolone;Drug: Sirolimus;Drug: PrednisonePrevail TherapeuticsEli Lilly and CompanyRecruiting18 Years50 YearsAll15Phase 1/Phase 2United States