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
No.TrialIDDate_
enrollment
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registration
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agemin
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agemax
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PhaseCountries
1NCT05208281
(ClinicalTrials.gov)
November 30, 202114/10/2021A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIMulticenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC GENERIUM, Russia) in Patients With Mucopolysaccharidosis Type IIMucopolysaccharidosis Type II;Metabolic DiseasesDrug: GNR-055 1.0-2.0-3.0 mg/kg;Drug: GNR-055 2.0 mg/kg;Drug: GNR-055 3.0 mg/kgAO GENERIUMNULLRecruitingN/AN/AMale32Phase 2/Phase 3Russian Federation