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Gene Therapy

CRISPR-Cas9 Modified Stem Cells for Sickle Cell Disease

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

Diagnosis of severe SCD as defined by documented SCD genotypes

History of at least two severe VOCs events per year for the previous two years prior to enrollment

Must not have

Clinically significant and active bacterial, viral, fungal, or parasitic infection

A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing a new treatment for sickle cell disease using a patient's own stem cells that have been modified with CRISPR-Cas9.

Who is the study for?

This trial is for children with severe Sickle Cell Disease who have had at least two serious pain episodes a year and haven't responded well to or can't tolerate Hydroxyurea treatment. They should be suitable for their own stem cell transplant, not have had one before, and not currently have any major infections.

What is being tested?

The study tests CTX001, which involves editing the patient's stem cells using CRISPR-Cas9 technology to treat severe SCD. It's an open-label trial meaning everyone knows they're getting this single-dose experimental therapy.

What are the potential side effects?

Potential side effects are likely related to the CRISPR-Cas9 process and stem cell transplant procedure but aren't specified here. Common risks may include immune reactions, infection risk from the procedure, and possible off-target genetic effects.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with severe sickle cell disease.

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I've had two or more severe pain crises a year for the last two years.

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I have tried hydroxyurea without success or cannot tolerate it.

Exclusion Criteria

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

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I do not have any active serious infections.

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

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I have had a stem cell transplant before.

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

Pivotal Trial

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

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

0 / 6Eligibility criteria met