Brief Title
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Official Title
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
Brief Summary
This is a single-dose, open-label study in pediatric participants with TDT. The study will
evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic
stem and progenitor cells (hHSPCs) (CTX001).
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
Secondary Outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Condition
Beta-Thalassemia
Intervention
CTX001
Study Arms / Comparison Groups
CTX001
Description: CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
12
Start Date
May 3, 2022
Completion Date
May 2026
Primary Completion Date
May 2026
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.
Gender
All
Ages
2 Years - 11 Years
Accepts Healthy Volunteers
No
Contacts
, 617-341-6777, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT05356195
Organization ID
VX21-CTX001-141
Secondary IDs
2021-002172-39
Responsible Party
Sponsor
Study Sponsor
Vertex Pharmaceuticals Incorporated
Collaborators
CRISPR Therapeutics
Study Sponsor
, ,
Verification Date
April 2022