This study evaluated the safety and efficacy of CTX001 in people with severe sickle cell disease (SCD). CTX001 involves collecting a person's own blood stem cells (autologous CD34+ hematopoietic stem and progenitor cells), modifying them in the laboratory with CRISPR-Cas9, and returning them as a single dose. The single-arm, open-label, multi-site Phase 1/2/3 study enrolled people with a documented severe SCD genotype, at least two severe vaso-occlusive crises per year over the previous two years, and eligibility for autologous stem cell transplant.
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 sickle cell disease as defined by: * Documented severe sickle cell disease genotype * History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment * Eligible for autologous stem cell transplant as per investigators judgment Key Exclusion Criteria: * An available 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.