Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Plain-language summary

This is a single-dose, open-label Phase 3 study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). It evaluates the safety and efficacy of CTX001 (exagamglogene autotemcel, exa-cel), made from the patient's own CD34+ human hematopoietic stem and progenitor cells modified with CRISPR-Cas9. Participants must be eligible for autologous stem cell transplant per investigator judgment, and those in the TDT group must have documented homozygous or compound heterozygous β-thalassemia.

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

Recruiting

Sponsor

Vertex Pharmaceuticals Incorporated

Intervention

CTX001 (Exagamglogene autotemcel, Exa-cel)

Start date

8/2/2022

Estimated completion

6/9/2027

Countries

United States, Germany, Italy, Saudi Arabia

Related genes

Locations

• New York, United States
• Charlotte, United States
• Nashville, United States
• Düsseldorf, Germany
• Rome, Italy
• Al Mathar Ash Shamali, Saudi Arabia

Eligibility criteria

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

Key Inclusion Criteria:

* Participants with TDT and SCD:
* Eligible for autologous stem cell transplant as per investigator's judgment.
* Participants with TDT:
* Diagnosis of TDT as defined by:
* 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
* History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
* Participants with SCD:
* Diagnosis of severe SCD as defined by:
* Documented SCD genotypes
* History of at least two severe VOCs events per year for the previous two years prior to enrollment

Key Exclusion Criteria:

* Participants with TDT and SCD:
* 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)
* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator
* Participants with TDT:
* Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications
* Participants with sickle cell β-thalassemia variant
* Participants with SCD:
* History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening

Other protocol defined Inclusion/Exclusion criteria may apply.