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

  
3 trials found
No.TrialIDDate_
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PhaseCountries
1JPRN-UMIN000023517
2016/10/0101/10/2016A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type CA study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C - IT-HPBCD therapy for NPC (Long-term) Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three yearsNara Medical UniversityNULLPending4years-old6years-oldMale and Female1Not selectedJapan
2JPRN-UMIN000020032
2014/12/1902/12/2015Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC)Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) - IT-HPBCD therapy in adult-onset NPC Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two yearsJichi Medical University, Saitama Medical CenterSaga UniversityKumamoto UniversityTohoku UniversityInstitute for Neurological DisordersComplete: follow-up continuing20years-oldNot applicableMale and Female2Not applicableJapan
3JPRN-UMIN000023094
2014/08/0110/07/2016A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type CA study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C - IT-HPBCD therapy for NPC Niemann-Pick disease type C2-hydroxypropyl-beta-cyclodextrin (HPBCD) 5-20mg/kg, every 4 weeks, for two yearsNara Medical UniversitySaga UniversityKumamoto UniversityComplete: follow-up continuing1years-old25years-oldMale and Female1Not selectedJapan