This Phase 3 study is a single-dose, open-label trial in pediatric participants with severe sickle cell disease (SCD) who have failed or cannot tolerate hydroxyurea. It evaluates the safety and efficacy of CTX001 (exagamglogene autotemcel, exa-cel), made from the patient's own CD34+ human hematopoietic stem and progenitor cells modified with CRISPR-Cas9. Key eligibility includes documented SCD genotypes, a history of at least two severe vaso-occlusive crises per year over the previous two years, and being eligible for autologous stem cell transplant per the investigator.
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 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 * Hydroxyurea (HU) failure unless HU intolerant * Eligible for autologous stem cell transplant as per investigators judgment Key Exclusion Criteria: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor * Prior hematopoietic stem cell transplant (HSCT). * Clinically significant and active bacterial, viral, fungal, or parasitic infection Other protocol defined Inclusion/Exclusion criteria may apply.