246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details


臨床試験数 : 23 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

  
1 trial found
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agemin
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1JPRN-jRCT2041210065
04/09/202104/09/2021A Phase I/II Study of Bucladesine in Patients with Severe Methylmalonic AcidemiaA Phase I/II Multi-Institutional Study of Bucladesine in Patients with Severe Methylmalonic Acidemia - McAMP methylmalonic acidemia;C537358Bucladesine will be administered intravenously over 60 minutes once a week. Starting dose is 0.3mg/kg. Dosage will be incerased to 0.55mg/kg, then 1.65mg/kg with monitoring PK and safety data.Ito TetsuyaNULLRecruiting>= 3month oldNot applicableBoth6Phase 1-2Japan