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Gene Therapy

Gene Transfer for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Erica Esrick, MD

Research Sponsored by David Williams

Eligibility Criteria Checklist

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

Must have

Age 3 years to 40 years

Failure of hydroxyurea therapy

Must not have

Contraindication to bone marrow harvest or conditioning medication

Uncontrolled infection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether using gene therapy to change the expression of a particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.

Who is the study for?

This trial is for people aged 3-40 with severe Sickle Cell Disease (SCD) who've had multiple pain events, acute chest syndrome, or need chronic transfusions. They must have tried hydroxyurea without success and can't have a matching bone marrow donor. Participants should be in stable health otherwise and willing to follow up for 15 years.

What is being tested?

The trial tests gene therapy using the patient's own modified blood stem cells to treat SCD. It involves a single infusion of these engineered cells aiming to increase fetal hemoglobin production which could potentially cure SCD.

What are the potential side effects?

Potential side effects may include reactions similar to those experienced during bone marrow transplants such as infection risk due to immune suppression from chemotherapy, discomfort at the infusion site, and possible organ inflammation.

Eligibility Criteria

Inclusion Criteria

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

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I am between 3 and 40 years old.

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Hydroxyurea treatment did not work for me.

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I don't have a family member who is a perfect match for a bone marrow donation.

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I have sickle cell disease with a specific genetic type.

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I have severe symptoms from sickle cell disease.

Exclusion Criteria

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

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I cannot undergo bone marrow harvest or take certain medications due to health reasons.

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I do not have any infections that aren't responding to treatment.

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I have a known bone marrow disorder or abnormal bone marrow cells.

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I have severe liver damage or hepatitis.

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I have had a transplant from a donor.

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I currently have cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 6 weeks post gene transfer then every 6 months until 2 years after gene transfer for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Rescue of hematopoiesis after conditioning

Secondary study objectives

Expression of transgene

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: Treatment armExperimental Treatment1 Intervention

open-label, non-randomized, single center, pilot and feasibility, single arm cohort study of a single infusion of autologous bone marrow derived CD34+ HSC cells transduced with lentiviral vector containing a short-hairpin RNA targeting BC11A.

0 / 11Eligibility criteria met