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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1JPRN-jRCT2031210279
15/09/202127/08/2021An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 Acid sphingomyelinase deficiencyDrug: Olipudase alfa (GZ402665)
Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion.
Tanaka TomoyukiNULLNot Recruiting>= 20age oldNot applicableBoth1Phase 2-3Japan
2NCT04877132
(ClinicalTrials.gov)
July 21, 20213/5/2021Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)Sphingomyelin LipidosisDrug: olipudase alfa (GZ402665)SanofiNULLApproved for marketing3 YearsN/AAllNULL