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

Part of paid clinical trials in New York, New York.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT05477563
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • CTX001 — BIOLOGICAL
    Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan

Study Details

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or 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.

Key Dates

Start date
Aug 2, 2022
Status verified
Mar 2026
Primary completion
Jun 9, 2027
Completion
Jun 9, 2027

Study Design

Enrollment
26 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CTX001
    CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter.

Primary Outcome Measure

Fetal Hemoglobin (HbF) Concentration Over Time [ Time Frame: Up to 12 Months After CTX001 Infusion ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew YorkNew York10032-
Levine Children's Hospital - HematologyCharlotteNorth Carolina28203-
TriStar Medical Group Children's Specialists - Pediatric OncologyNashvilleTennessee37203-

Find similar trials in New York, NY

By condition
Sickle cell disease
By research site
New York Presbyterian Hospital - Morgan Stanley Children's Hospital· New York, NYLevine Children's Hospital - Hematology· Charlotte, NCTriStar Medical Group Children's Specialists - Pediatric Oncology· Nashville, TN

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