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

  
1 trial found
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1JPRN-UMIN000009758
2013/06/2415/01/2013Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I studyDevelopment of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study - Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome)Treosulfan 14 g/m2, intravenous,
day -6 -5 -4
Tokai University School of Medicine1. School of Human Health Science Faculty of Medicine Kyoto University2. Department of Pediatrics, Japanese Red cross Nagoya Daiichi Hospital3. Department of Pediatrics, Nihon UniversityComplete: follow-up completeNot applicableNot applicableMale and Female12Phase 1Japan