Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Sponsored by Vertex Pharmaceuticals Incorporated
About this trial
Last updated 2 years ago
Study ID
Status
Type
Phase
Placebo
Accepting
Not accepting
Trial Timing
Started 3 years ago
What is this trial about?
What are the Participation Requirements?
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.