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

Plain-language summary

This Phase 3 study is a single-dose, open-label trial in pediatric participants with transfusion-dependent β-thalassemia (TDT). It evaluates the safety and efficacy of CTX001 (exagamglogene autotemcel, exa-cel), autologous CD34+ human hematopoietic stem and progenitor cells modified with CRISPR-Cas9. Participants must have documented homozygous or compound heterozygous β-thalassemia, including β-thalassemia/hemoglobin E (HbE); enrollment may be based on historical data, but the genotype is confirmed by the study central laboratory.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Active, not recruiting

Sponsor

Vertex Pharmaceuticals Incorporated

Intervention

CTX001 (Exagamglogene autotemcel, Exa-cel)

Start date

5/3/2022

Estimated completion

11/14/2027

Countries

United States, Canada, Germany, Italy, United Kingdom

Related genes

Locations

• Nashville, United States
• Toronto, Canada
• Düsseldorf, Germany
• Rome, Italy
• London, United Kingdom
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.