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. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCTs051180113 | 01/04/2019 | 11/03/2019 | Novel replacement therapy for inherited GPI deficiency | Novel 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 Shin | NULL | Recruiting | Not applicable | Not applicable | Both | 5 | N/A | Japan |
| 2 | JPRN-UMIN000030314 | 2019/04/01 | 15/01/2018 | Novel 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 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. | Osaka University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 5 | Not selected | Japan |
| 3 | JPRN-UMIN000024185 | 2014/07/31 | 27/09/2016 | Prospective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiency | Prospective 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 deficiency | Initial 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 University | Department of Child Neurology, Tottori University Hospital | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan |