跳至主要内容
临床试验/JPRN-jRCTs021200008
JPRN-jRCTs021200008已完成2 期

Phase II study of galactosidase as therapy for type IV galactosemia

Kure Shigeo0 个研究点目标入组 3 人开始时间: 2020年6月8日最近更新:
适应症

试验速览

阶段
2 期
状态
已完成
发起方
入组人数
3

研究概览

简要总结

This study demonstrated that the galactosidase treatment for type IV galactosemia can reduce blood galactose levels in lactose loading, suggesting the galactosidase may be a therapeutic agent for type IV galactosemia.

研究设计

研究类型
Interventional

入排标准

年龄范围
>= 1age old 至 < 10age old(—)
性别
All

入选标准

  • Patients are diagnosed to type IV galactosemia by genetic test (pathogenic variants in GALM gene are identified by exome or direct sequencing) or GALM enzyme activity test.

排除标准

  • (1) Patients with severe liver disease or liver injury (AST, ALT > 1.25 x upper limits of normal)
  • (2) Patients have participated to other clinical study or trial
  • (3) Patients with constipation which is in need of treat
  • (4) Patients with psychological problem

研究者

发起方
Kure Shigeo

相似试验