320. 先天性グリコシルホスファチジルイノシトール(GPI)欠損症 Inherited glycosylphosphatidylinositol deficiency Clinical trials / Disease details


臨床試験数 : 3 薬物数 : 2 - (DrugBank : 2) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
2 trials found
No.TrialIDDate_
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agemin
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agemax
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PhaseCountries
1JPRN-jRCTs051180113
01/04/201911/03/2019Novel replacement therapy for inherited GPI deficiencyNovel replacement therapy for inherited GPI deficiency - Novel replacement therapy for inherited GPI deficiency Inherited GPI deficiency
Inherited GPI deficiency
Folinic acid 1mg/kg/day in three divided dose to start. If the 5-MTHF concentration in cerebrospinal fluid will be low after one month, folinic acid will be uo to 2-5mg/kg/day.The period of administration is six months in principle.Nabatame ShinNULLRecruitingNot applicableNot applicableBoth5N/AJapan
2JPRN-UMIN000030314
2019/04/0115/01/2018Novel replacement therapy for inherited GPI deficiency (IGD)Novel replacement therapy for inherited GPI deficiency (IGD) - Novel replacement therapy for inherited GPI deficiency (IGD) Inherited GPI deficiencyFolinic acid 1mg/kg/day in three divided dose to start. If the 5-MTHF concentration in cerebrospinal fluid will be low after one month, folinic acid will be uo to 2-5mg/kg/day.The period of administration is six months in principle.Osaka UniversityNULLRecruitingNot applicableNot applicableMale and Female5Not selectedJapan