EUCTR2021-002173-26-DE进行中（未招募）1 期
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Subjects 2 through 11 years of age, inclusive, on the date of informed consent
- •Diagnosis of severe sickle cell disease as defined by:
- •Documented severe sickle cell disease genotype
- •History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment
- •Eligible for autologous stem cell transplant as per investigators judgment
- •Other protocol defined inclusion criteria may apply
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •An available 10/10 human leukocyte antigen (HLA)-matched related donor
- •Prior hematopoietic stem cell transplant (HSCT)
- •Clinically significant and active bacterial, viral, fungal, or parasitic infection
- •Other protocol defined exclusion criteria may apply
研究者
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