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
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
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