2024-514641-12-00招募中3 期
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- HbF and Hb levels over time. The evaluation will start 60 days after last RBC transfusion for post-transplant support or disease management.
研究概览
简要总结
To assess HbF levels over time, after a single dose of autologous CRISPR/Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs)(CTX001) in adolescent and adult subjects with either Transfusion dependent β-Thalassemia (TDT) or severe sickle cell disease (SCD)
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Participants with TDT and SCD: Eligible for autologous stem cell transplant as per investigator's judgment.
- •Participants with TDT: Diagnosis of TDT as defined by: Documented homozygous β-thalassemia 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 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
- •Participants with SCD: Diagnosis of severe SCD as defined by: Documented SCD genotypes History of at least two severe VOCs events per year for the previous two years prior to enrollment
排除标准
- •Participants with TDT and SCD: 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) Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
- •Participants with TDT: Participants with associated α-thalassemia and >1 alpha deletion, or alpha multiplications Participants with sickle cell β-thalassemia variant
- •Participants with SCD: History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening
结局指标
主要结局
HbF and Hb levels over time. The evaluation will start 60 days after last RBC transfusion for post-transplant support or disease management.
HbF and Hb levels over time. The evaluation will start 60 days after last RBC transfusion for post-transplant support or disease management.
次要结局
- TDT and SCD: Safety and tolerability of CTX001 based on adverse events (AEs), clinical laboratory values, neutrophil engraftment, platelet engraftment, transplant-related mortality (TRM), and all-cause mortality.
- TDT and SCD: Relative reduction from baseline in annualized volume of RBC transfusions
- TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time
- TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time
- TDT: Duration transfusion free
- SCD: Relative reduction from baseline in annualized rate of severe vaso-occlusive crises (VOCs) up to 12 months after CTX001 infusion. The evaluation starts 60 days after last RBC transfusion for post-transplant support or SCD disease management.
- SCD: Relative reduction from baseline in annualized rate of inpatient hospitalizations for severe VOCs up to 12 months after CTX001 infusion. The evaluation starts 60 days after last RBC transfusion for post-transplant support or SCD disease management.
- SCD: Relative reduction from baseline in annualized duration of hospitalization for severe VOCs up to 12 months after CTX001 infusion. The evaluation starts 60 days after last RBC transfusion for post-transplant support or SCD disease management.
- SCD: Relative reduction from baseline in markers of hemolysis up to 12 months after CTX001 infusion.
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.
研究点 (2)
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