Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Vertex Pharmaceuticals

Status and phase: Begins enrollment in a year or more; Phase 3

Conditions: Sickle Cell Disease

Treatments: Biological: Exa-cel

Study type: Interventional

Funder types: Industry

Identifiers

NCT05951205

2021-006375-41 (EudraCT Number)

VX21-CTX001-171

2023-503247-34-00 (Other Identifier)

Details and patient eligibility

About

The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).

Enrollment: 12 estimated patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants with documented βS/βC (HbSC) genotype
• Participants must be 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
• Participants with prior hematopoietic stem cell transplant (HSCT)
• Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Exa-cel

Experimental group

Description:

Participants will receive a single infusion of exa-cel (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene) through a central venous catheter.

Treatment:

Biological: Exa-cel