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

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13 - 40

Phase 2

9 Locations

NCT05353647

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

Contacts


Leah Cheng, 857-218-4731, leah.cheng@childrens.harvard.edu

Lauren Holmes, graspstudy-dl@childrens.harvard.edu

Locations

Children's Hospital of Los AngelesRECRUITING

Los Angeles, California
Contacts:

UC Davis Medical CenterRECRUITING

Sacramento, California
Contacts:

UCLA Medical CenterRECRUITING

Los Angeles, California
Contacts:

UCSF Benioff Children's Hospital OaklandRECRUITING

Oakland, California
Contacts:

Children's Healthcare of Atlanta/Emory UniversityACTIVE_NOT_RECRUITING

Atlanta, Georgia

Lurie Children's Hospital of ChicagoACTIVE_NOT_RECRUITING

Chicago, Illinois

Boston Children's HospitalACTIVE_NOT_RECRUITING

Boston, Massachusetts

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

Boston, Massachusetts

Medical College of WisconsinACTIVE_NOT_RECRUITING

Milwaukee, Wisconsin

ClinicalTrials.gov record


NCT05353647. First posted on 4/29/22

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