NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
| NCT ID | NCT05477563 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Condition | Beta-Thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 26 participants |
| Start Date | 2022-08-02 |
| Primary Completion | 2027-06-09 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Eligibility Criteria
Key Inclusion Criteria: * 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 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 * 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 Key Exclusion Criteria: * Participants with TDT and SCD: * A willing and healthy 10