CTX001 for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+17 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 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.

Eligibility Criteria

This trial is for people with severe sickle cell disease, who've had at least two major pain crises a year over the last two years. They must be suitable for their own stem cell transplant but can't have an available perfect-match donor or previous transplants. Active serious infections disqualify them.

Inclusion Criteria

I have severe sickle cell disease with at least two major pain crises per year.

Exclusion Criteria

I have a perfect match for a stem cell donor and no active infections.

Participant Groups

The study tests CTX001, which uses CRISPR-Cas9 to modify patients' own blood-forming cells aiming to treat severe sickle cell disease. It's a single-arm trial where all participants receive one dose of this gene-editing therapy and are monitored for safety and effectiveness.

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). Subjects 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: