CRISPR-Cas9 Gene Editing for Beta Thalassemia

Recruiting in Palo Alto (17 mi)

+21 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Vertex Pharmaceuticals Incorporated

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Eligibility Criteria

This trial is for individuals with transfusion-dependent β-thalassemia, which means they need regular blood transfusions due to their condition. They should have a history of significant blood transfusion needs and be suitable for an autologous stem cell transplant. People with certain genetic variations of thalassemia or active infections, low white blood cell or platelet counts can't participate.

Inclusion Criteria

I've had significant blood transfusions in the last 2 years.

I am considered a candidate for a stem cell transplant using my own cells.

I have been diagnosed with transfusion-dependent β-thalassemia.

Exclusion Criteria

White blood cell (WBC) count <3 × 10^9/L or platelet count <50 × 10^9/L not related to hypersplenism

I have a healthy, fully matched donor for my treatment.

I have had a stem cell transplant from a donor.

+3 more

Participant Groups

The study is testing CTX001, which involves modifying the patient's own stem cells using CRISPR-Cas9 technology to potentially treat β-thalassemia. It's a single-dose study looking at both safety and how well it works in improving the condition.

1Treatment groups

Experimental Treatment

Group I: CTX001Experimental Treatment1 Intervention

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.

CTX001 is already approved in European Union, United States for the following indications:

🇪🇺 Approved in European Union as CTX001 for: