180. ATR-X syndrome Clinical trials / Disease details


Clinical trial : 1 Drugs : 3 - (DrugBank : 2) / Drug target gene : 0 - Drug target pathway : 0

  
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1JPRN-jRCT2051220062
14/07/202214/07/2022Physician-initiated clinical trial in patients with 5-aminolevulinic acid-induced ATR-X syndrome.Exploratory study to investigate the safety and efficacy of NPJ005 in patients with ATR-X syndrome. - IACT21003 ATR-X syndrome
infant, intellectual disability, epigenetics, guanine quadruplex;D008607
Containing 5-aminolevulinic acid (5-ALA) hydrochloride and sodium ferrous citrate (SFC)
Tablets (25 mg 5-ALA hydrochloride/39.2 mg SFC per tablet, NPJ005) for 24 weeks
Wada TakahitoNULLRecruiting>= 2age oldNot applicableMale5Phase 2Japan