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This single-arm, open-label, multi-site, single-dose study evaluates CTX001 (exagamglogene autotemcel, exa-cel) in participants with transfusion-dependent β-thalassemia (TDT). The treatment uses the patient's own CD34+ human hematopoietic stem and progenitor cells modified with CRISPR-Cas9, and the trial is conducted under a Phase 2/3 designation. Eligibility requires documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE); participants can be enrolled on historical data, but genotype confirmation is required.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by 1. 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 2. History of at least 100 mL/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 * Eligible for autologous stem cell transplant as per investigator's judgment Key Exclusion Criteria: * A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is available per investigator's judgement * Prior allo-HSCT * Participants with associated α-thalassemia and \>1 alpha deletion or alpha multiplications * Participants 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 \<50 × 10\^9/L not related to hypersplenism Other protocol defined Inclusion/Exclusion criteria may apply.