Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Vertex Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: Hydroxyurea Failure; Hemoglobinopathies; Hydroxyurea Intolerance; Hematological Diseases; Sickle Cell Disease

Treatments: Biological: CTX001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05329649

VX21-CTX001-151

2021-002173-26 (EudraCT Number)

Details and patient eligibility

About

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Enrollment: 15 estimated patients

Sex: All

Ages: 2 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• 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
• Hydroxyurea (HU) failure unless HU intolerant
• Eligible for autologous stem cell transplant as per investigators judgment

Key Exclusion Criteria:

• A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor
• Prior hematopoietic stem cell transplant (HSCT).
• Clinically significant and active bacterial, viral, fungal, or parasitic infection

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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15 participants in 1 patient group

CTX001

Experimental group

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.

Treatment:

Biological: CTX001