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 - VX21-CTX001-161

Vertex Pharmaceuticals Inc.0 个研究点目标入组 22 人开始时间: 2024年6月27日最近更新: 2024年7月22日

试验速览

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•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 ß-thalassemia or compound heterozygous ß-thalassemia including ß-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

•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 a-thalassemia and >1 alpha deletion, or alpha multiplications Participants with sickle cell ß-thalassemia variant, Participants with SCD: History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening

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