A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thalassemia

Vertex Pharmaceuticals Incorporated0 个研究点目标入组 15 人开始时间: 2021年10月1日最近更新: 2024年8月5日

适应症

试验速览

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•Subjects 2 through 11 years of age, inclusive, on the date of informed consent.
•Diagnosis of transfusion-dependent ß-thalassemia (TDT) as defined by:
•a. Documented homozygous 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 start of busulfan conditioning.
•b. History of at least 100 mL/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 subjects 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.
•Other protocol defined inclusion criteria may apply.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 15
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•An available 10/10 Human Leukocyte Antigen (HLA)-matched related donor.
•Prior allo-HSCT.
•Subjects with associated a-thalassemia and >1 alpha chain deletion or alpha multiplications.
•Subjects with sickle cell beta thalassemia variant.
•Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
•White blood cell (WBC) count <3 × 10^9/L or platelet count <150 × 10^9/L not related to hypersplenism.
•Other protocol defined exclusion criteria may apply.

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