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 ß-Thalassemia or Severe Sickle Cell Disease

Vertex Pharmaceuticals Incorporated0 个研究点目标入组 26 人2022年3月8日

概览

阶段: 1 期
干预措施: 未指定
疾病 / 适应症: Transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD)
发起方: Vertex Pharmaceuticals Incorporated
入组人数: 26
状态: 进行中（未招募）
最后更新: 去年

暂无简介。

注册库

who.int

开始日期

2022年3月8日

结束日期

待定

最后更新

去年

研究类型

Interventional clinical trial of medicinal product

性别

All

发起方

•Subjects with TDT:
•Subjects 12 to 35 years of age, inclusive, on the date of informed consent
•Eligible for autologous stem cell transplant as per investigator’s judgement
•Diagnosis of transfusion\-dependent ß\-thalassemia (TDT) as defined by:
•Documented homozygous ß\-thalassemia or compound heterozygous ß\-thalassemia including ß\-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning.
•A history of at least 100mL/kg/year or 10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re\-screening
•Subjects with SCD:
•Subjects 12 to 35 years of age, inclusive, on the date of informed consent
•Documented ßS/ßS, ßS/ß0, or ßS/ß\+, subjects can be enrolled based on historical genotype results, but confirmation of genotype is required before busulfan conditioning
•Eligible for autologous stem cell transplant as per investigator’s judgment

•Subjects with TDT:
•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).
•Subjects with associated a\-thalassemia and \>1 alpha deletion, or alpha multiplications.
•Subjects with sickle cell ß\-thalassemia variant.
•Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
•White blood cell (WBC) count \<3 × 109/L or platelet count \<50 × 109/L not related to hypersplenism per investigator judgment.
•Subjects with SCD:
•A willing and healthy 10/10 Human Leukocyte Antigen (HLA)\-matched related donor is available per investigator’s judgement.