跳至主要内容
临床试验/NL-OMON42151
NL-OMON42151已完成不适用

Evaluation of the ex vivo effect of AG-348 treatment on pyruvate kinase-deficient cell metabolism and erythroid development - Ex vivo effect of AG-348 on PKD

Agios Pharmaceuticals Inc.0 个研究点目标入组 30 人开始时间: 待定最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
30

研究概览

简要总结

暂无简介。

研究设计

研究类型
Observational

入排标准

年龄范围
18 至 99(—)

入选标准

  • 1. Adult patients with biochemically and genetically diagnosed PK deficiency, i.e. patients should be compound heterozygous or homozygous for mutations in PKLR, the gene that encodes red blood cell PK.
  • 2. The participant is willing and able to give written informed consent.

排除标准

  • Patients who have been recently transfused with packed red blood cells (*Recently* is defined as less than 3 months before blood collection).

研究者

相似试验