2024-513349-35-00招募中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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Proportion of subjects who achieve TI12. A subject will be considered to have achieved TI12 if he/she has maintained weighted average Hb ≥9 g/dL without RBC transfusions for at least 12 consecutive months any time after CTX001 infusion. The evaluation of TI12 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.
研究概览
简要总结
Evaluate the efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001) in pediatric subjects with transfusion-dependent β-thalassemia (TDT)
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of TDT as defined by: a. 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. b. 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.
结局指标
主要结局
Proportion of subjects who achieve TI12. A subject will be considered to have achieved TI12 if he/she has maintained weighted average Hb ≥9 g/dL without RBC transfusions for at least 12 consecutive months any time after CTX001 infusion. The evaluation of TI12 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.
Proportion of subjects who achieve TI12. A subject will be considered to have achieved TI12 if he/she has maintained weighted average Hb ≥9 g/dL without RBC transfusions for at least 12 consecutive months any time after CTX001 infusion. The evaluation of TI12 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.
次要结局
- Proportion of Participants Achieving at Least 95 Percent (%), 90%, 85%, 75% and 50% Reduction in Annualized Transfusions
- Relative Reduction in Annualized Volume of RBC Transfusions
- Transfusion Free Duration for Participants who Achieve TI12
- Proportion of Alleles With Intended Genetic Modification Present in Peripheral Blood Over Time
- Proportion of Alleles With Intended Genetic Modification Present in CD34+ Cells of the Bone Marrow Over Time
- HbF concentration (pre-transfusion) over time
- Total hemoglobin concentration (pre-transfusion) over time.
- Safety and tolerability of CTX001 based on adverse events (AEs), clinical laboratory values, vital signs, neutrophil engraftment, platelet engraftment, transplant-related mortality (TRM), and all-cause mortality
- Proportion of subjects who achieve TI6. A subject will be considered to have achieved TI6 if he/she has maintained weighted average Hb ≥9 g/dL without RBC transfusions for at least 6 consecutive months any time after CTX001 infusion. The evaluation of TI6 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.
研究点 (2)
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