Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Apr 29, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called CTX001 for children with a serious blood disorder known as transfusion-dependent beta-thalassemia (TDT). TDT is a condition where the body does not produce enough healthy red blood cells, leading to the need for regular blood transfusions. The trial will test the safety and effectiveness of using modified stem cells from the patient’s own body, which have been altered using a technology called CRISPR-Cas9 to help produce healthy blood cells.

To be eligible for this study, participants need to have a confirmed diagnosis of TDT and a history of needing regular blood transfusions over the past two years. The trial is open to children aged 2 to 17, regardless of gender. Participants will receive a single dose of CTX001, and the study team will closely monitor their health and the treatment's effects. It's important to note that this trial is specifically for those who do not have a suitable family member donor for a traditional stem cell transplant and have not undergone any prior stem cell transplants. This study aims to provide a potential new option for improving the health and quality of life for children affected by this condition.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• * Diagnosis of TDT as defined by:
• • Documented homozygous 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 mL/kilograms (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 participants 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.
• Key Exclusion Criteria:
• • A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement
• • Prior hematopoietic stem cell transplant (HSCT)
• • Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
• • Participants with sickle cell β-thalassemia variant
• • Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
• • Other protocol defined Inclusion/Exclusion criteria may apply.