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Gene therapy using your own cells to treat sickle cell disease (SCD) in teens and young adults 


13 - 40


9 Locations


Clinical Trial Goal

To find out if gene therapy is safe and works well to treat SCD in teens and young adults

You may be able to join this trial if you:

  • Are 13 – 40 years old
  • Have SCD with genotype HbSS or HbS/beta thalassemia
  • Had 4 or more vaso-occlusive events in the last 2 years. This may include pain crises, stroke, acute chest syndrome, and other complications
  • Do not have someone who matches you and can be your donor for an allogeneic (cells from a donor) blood or marrow transplant (BMT)
  • Have not had an allogeneic BMT
  • Agree to have other standard tests done to see if you can be in the clinical trial 

Trial Details

First, you'll get blood transfusions for at least 3 months. Then you'll have apheresis, a process to collect, separate and save your blood-forming cells.

Doctors will treat your blood-forming cells in a lab with gene therapy. The therapy will change the BCL11A gene in the blood-forming cells to help it make more fetal hemoglobin. Fetal hemoglobin is a healthy type of hemoglobin that doesn't sickle.

You'll get standard chemotherapy (chemo) with busulfan to make room for the blood-forming cells

Then, your treated blood-forming cells are given back to you through an intravenous (IV) infusion.

You'll have tests to see how well the treatment is working. The clinical trial doctors will check your health for 2 years.

The Food and Drug Administration (FDA) has not yet approved this gene therapy for SCD.

Watch a video about gene therapy


Leah Cheng, 857-218-4731,

Catherine Dempsey,


Children's Hospital of Los AngelesRECRUITING

Los Angeles, California
Neena Kapoor, MD,

UC Davis Medical CenterRECRUITING

Sacramento, California
Mehrdad Abedi, MD,


Los Angeles, California
Gary Schiller, MD,

UCSF Benioff Children's Hospital OaklandRECRUITING

Oakland, California
Mark Walters, MD,

Children's Healthcare of Atlanta/Emory UniversityRECRUITING

Atlanta, Georgia
Edmund Waller, MD,

Lurie Children's Hospital of ChicagoRECRUITING

Chicago, Illinois
Sonali Chaudhury, MD,

Boston Children's HospitalRECRUITING

Boston, Massachusetts

Dana-Farber Cancer Institute/Brigham and Women's HospitalRECRUITING

Boston, Massachusetts
Joseph Antin, MD,

Medical College of WisconsinRECRUITING

Milwaukee, Wisconsin
Mary Eapen, MD, MS, 414-805-0700, record

NCT05353647. First posted on 4/29/22

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