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
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
Catherine Dempsey, graspstudy-dl@childrens.harvard.edu
Leah Cheng, 857-218-4731, leah.cheng@childrens.harvard.edu
Locations
Sponsors
lead: David Williams, collaborator: National Heart, Lung, and Blood Institute (NHLBI), collaborator: California Institute for Regenerative Medicine (CIRM), collaborator: bluebird bio, collaborator: Blood and Marrow Transplant Clinical Trials Network
