← Back to Search

Gene Therapy

CRISPR-Cas9 Gene Editing for Beta Thalassemia

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

History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening

Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning

Must not have

Prior allo-HSCT

Subjects with associated α-thalassemia and >1 alpha deletion or alpha multiplications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening visit through month 24 visit

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment for people with a blood disorder called transfusion-dependent β-thalassemia. The treatment involves using CRISPR-Cas9 to modify the patient's own blood cells. The trial will evaluate the safety and efficacy of this treatment.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not specified here but generally may include reactions related to the modification process of stem cells, body's response to genetically edited cells, and typical risks associated with stem cell transplants such as infection risk.

Eligibility Criteria

Inclusion Criteria

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

Select...

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

Select...

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have had a stem cell transplant from a donor.

Select...

I have α-thalassemia with more than one alpha gene change.

Select...

I have the sickle cell beta thalassemia variant.

Select...

I do not have any serious or active infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening visit through month 24 visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening visit through month 24 visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening visit through month 24 visit 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 health-related quality of life (HRQoL) from baseline over time using EuroQol Questionnaire (5 dimensions - 5 levels of severity - EQ-5D-5L)

Change in health-related quality of life (HRQoL) from baseline over time using the Functional assessment of cancer therapy-bone marrow transplant questionnaire (FACT-BMT)

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 / 6Eligibility criteria met