Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Vertex Pharmaceuticals Incorporated
With
CRISPR Therapeutics
Age
2–11
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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
Clareo Health | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
