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

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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-UMIN000023517	2016/10/01	01/10/2016	A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann- ...	A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann- ...	Niemann-Pick disease type C	2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years	Nara Medical University	NULL	Pending	4years-old	6years-old	Male and Female	1	Not selected	Japan
2	JPRN-UMIN000020032	2014/12/19	02/12/2015	Intrathecal 2-hydroxypropyl-beta-cyclodextrin(HPBCD) therapy in adult-onset Niemann-Pick disease typ ...	Intrathecal 2-hydroxypropyl-beta-cyclodextrin(HPBCD) therapy in adult-onset Niemann-Pick disease typ ...	Niemann-Pick disease type C	2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two years	Jichi Medical University, Saitama Medical Center	Saga UniversityKumamoto UniversityTohoku UniversityInstitute for Neurological Disorders	Complete: follow-up continuing	20years-old	Not applicable	Male and Female	2	Not applicable	Japan
3	JPRN-UMIN000023094	2014/08/01	10/07/2016	A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick diseas ...	A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick diseas ...	Niemann-Pick disease type C	2-hydroxypropyl-beta-cyclodextrin (HPBCD) 5-20mg/kg, every 4 weeks, for two years	Nara Medical University	Saga UniversityKumamoto University	Complete: follow-up continuing	1years-old	25years-old	Male and Female	1	Not selected	Japan

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