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Gene Therapy

CRISPR Therapy (CTX001) for Thalassemia

Phase 3

Recruiting

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/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy <24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months

Be younger than 18 years old

Must not have

Prior hematopoietic stem cell transplant (HSCT)

Participants with sickle cell β-thalassemia variant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.

Who is the study for?

This trial is for children with Transfusion-Dependent β-Thalassemia (TDT) who need regular blood transfusions and are suitable for a stem cell transplant. They must have specific genetic forms of TDT confirmed by the study's lab. Those with a perfect match donor, previous transplants, certain sickle cell disease variants, or active infections can't participate.

What is being tested?

The trial tests CTX001, which involves editing patients' own stem cells using CRISPR-Cas9 technology to potentially treat TDT. It's an open-label study where all participants receive one dose of the modified cells after a conditioning regimen.

What are the potential side effects?

Potential side effects may include reactions related to the stem cell transplant process, immune responses against edited cells, and complications from the gene-editing procedure itself. The exact side effects will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I have needed regular blood transfusions for at least 6 months.

Exclusion Criteria

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

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I have had a stem cell transplant before.

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I have the sickle cell β-thalassemia variant.

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

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I do not have any serious or active infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

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

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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). Participants will receive single infusion of CTX001 through central venous catheter.

0 / 5Eligibility criteria met