CTX001 for Sickle Cell Disease

Phase 2 & 3

Waitlist Available

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 months up to 2 years after ctx001 infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a possible new treatment for sickle cell disease using CRISPR-Cas9 technology to modify patients' own stem cells. The goal is to improve safety and efficacy compared to current treatments.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include reactions related to stem cell transplantation, such as immune system complications, infection risks, and possible off-target genetic effects due to the CRISPR-Cas9 editing process.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 months up to 2 years after ctx001 infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 months up to 2 years after ctx001 infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 months up to 2 years after ctx001 infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in PRO over time assessed using EuroQol quality of life scale (EQ-5D-5L)

Change in PRO over time assessed using adult sickle cell quality of life measurement system (ASCQ-Me)

Change in PRO over time assessed using functional assessment of cancer therapy-bone marrow transplant (FACT-BMT) questionnaire

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

0 / 1Eligibility criteria met