NL-OMON51728招募中不适用
Targeting Pyruvate kinase as a therapeUtic option in rare aneMiA - PUMA-Study - PUMA-project
niversitair Medisch Centrum Utrecht0 个研究点目标入组 115 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
- 招募中
- 发起方
- 入组人数
- 115
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
- 16 至 99（—）
入选标准
- •1. Participant is diagnosed with one of following diseases: sickle cell
- •disease, hereditary spherocytosis, hereditary xerocytosis, red blood cell
- •enzymopathy, thalassemia, Diamond-Blackfan anemia, congenital dyserythropoietic
- •anemia or myelodysplastic syndrome.
- •2. Participant (or legal guardian) is willing and able to give informed
排除标准
- •1. Children (aged under 16 years).
- •2. Transfusion-dependence (defined as more than 12 transfusions a year).*
- •3. Recent transfusion (defined as within 3 months prior to enrolment).**
- •4. Other concomitant RBC diseases.
- •5. Currently receiving chemotherapeutics.
- •6. Currently receiving experimental treatment in the context of a clinical
- •* For thalassemia this will not be a criterion since we will also include
- •patients who are on chronic transfusion therapy. Since the severe patients will
- •be treated with blood transfusion.
- •**For DBA, this will not be a criterion. As DBA is extremely rare, this
- •exclusion criterion may lead to insufficient eligible participants.
研究者
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