A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Vertex Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3; Phase 2

Conditions: Hemoglobinopathies; Hematological Diseases; Sickle Cell Disease

Treatments: Biological: CTX001

Study type: Interventional

Funder types: Industry

Identifiers

NCT03745287

CTX001-121

Details and patient eligibility

About

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Enrollment: 63 patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Diagnosis of severe sickle cell disease as defined by:
• Documented severe sickle cell disease genotype
• History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment
• Eligible for autologous stem cell transplant as per investigators judgment

Key Exclusion Criteria:

• An available 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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63 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). Subjects will receive a single infusion of CTX001 through a central venous catheter.

Treatment:

Biological: CTX001