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

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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


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)





Study Arms / Comparison Groups

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.


* 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


Estimated Enrollment


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

          -  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.




2 Years - 11 Years

Accepts Healthy Volunteers



, 617-341-6777, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Vertex Pharmaceuticals Incorporated


 CRISPR Therapeutics

Study Sponsor

, , 

Verification Date

April 2022