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

  
No.TrialIDDate_
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agemin
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PhaseCountries
1JPRN-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
2JPRN-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
3JPRN-UMIN000024185
2014/07/3127/09/2016Prospective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiencyProspective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiency - Interventional study of vitamin B6 for inherited GPI deficiency Inherited GPI deficiencyInitial dose: pyridoxine 10-15 mg/kg/day
1-2 weeks after the initiation, the dose is increased to maintenance dose, if there is no adverse effect.
Maintenance dose: pyridoxine 20-30 mg/kg/day
We prescribe pyridoxine for a year.
The Graduate School of Medicine, Osaka UniversityDepartment of Child Neurology, Tottori University HospitalComplete: follow-up completeNot applicableNot applicableMale and Female15Not selectedJapan