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
12–35
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by
1. 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
2. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
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 allo-HSCT
Participants with associated α-thalassemia and \>1 alpha deletion or alpha multiplications
Participants with sickle cell beta thalassemia variant
Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
White blood cell (WBC) count \<3 × 10\^9/L or platelet count \<50 × 10\^9/L not related to hypersplenism
Clareo Health | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
