NCT05356195进行中（未招募）3 期
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
研究概览
简要总结
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of TDT as defined by:
- •Documented homozygous or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning
- •History of at least 100 mL/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy <24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months
- •Eligible for autologous stem cell transplant as per investigator's judgment.
排除标准
- •A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
- •Prior hematopoietic stem cell transplant (HSCT)
- •Participants with associated α-thalassemia and >1 alpha deletion, or alpha multiplications
- •Participants with sickle cell β-thalassemia variant
- •Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
CTX001
Experimental
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.
干预措施: CTX001 (Biological)
结局指标
主要结局
Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
时间窗: Up to 24 Months After CTX001 Infusion
次要结局
- Proportion of Participants Achieving at Least 95 Percent (%), 90%, 85%, 75% and 50% Reduction in Annualized Transfusions(From Baseline up to 24 Months After CTX001 Infusion)
- Transfusion Free Duration for Participants who Achieve TI12(Up to 24 Months After CTX001 Infusion)
- Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time(Up to 24 Months After CTX001 Infusion)
- Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time(Up to 24 Months After CTX001 Infusion)
- Change in Fetal Hemoglobin Concentration Over Time(From Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion)
- Change in Total Hemoglobin Concentration Over Time(From Baseline (Pre-transfusion) up to 24 Months After CTX001 Infusion)
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(From Signing of Informed Consent up to 24 Months After CTX001 Infusion)
- Proportion of Participants With Engraftment (First day of 3 Consecutive Measurements of Absolute Neutrophil Count [ANC] ≥500 per Microliter [mcgL] on 3 Different Days)(Within 42 Days After CTX001 Infusion)
- Time to Engraftment(Up to 24 Months After CTX001 Infusion)
- Incidence of Transplant-related Mortality (TRM) Within 100 Days After CTX001 Infusion(Within 100 Days After CTX001 Infusion)
- Incidence of TRM Within 12 Months After CTX001 Infusion(Within 12 Months After Infusion)
- Relative Reduction in Annualized Volume and Episodes of RBC Transfusions starting Month 10 After CTX001 infusion(From Baseline up to 24 Months After CTX001 Infusion)
- Incidence of All-cause Mortality(From Signing of Informed Consent up to 24 Months After CTX001 Infusion)
研究者
研究点 (6)
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