Clinical Trial Goal
To find out if gene therapy, ALS20, is safe and works well to treat transfusion-dependent beta thalassemia
You may be able to join this trial if you:
- Are 18 - 35 years old
- Have had at least 8 transfusions in the last 2 years
- Do not have a family member who is a match for you and can be your donor
- Have not been treated with gene therapy
- Have not had a blood or marrow transplant (BMT)
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
ALS20 is a lentiviral vector that helps deliver a healthy beta-goblin gene into your blood stem cells. Doctors think this will help your body produce red blood cells with normal hemoglobin.
First, 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 ALS20. The therapy will add the beta goblin gene to the blood-forming cells to help decrease the need for blood transfusions.
Then, your treated blood-forming cells are given back to you through an intravenous (IV) infusion.
The clinical trial doctors will check your health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved ALS20.
Watch a video about gene therapy
Doctors will treat your blood-forming cells in a lab with ALS20. The therapy will add the beta goblin gene to the blood-forming cells to help decrease the need for blood transfusions.
Then, your treated blood-forming cells are given back to you through an intravenous (IV) infusion.
The clinical trial doctors will check your health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved ALS20.
Watch a video about gene therapy
Contacts
Janet Kwiatkowski, MD, 215-590-5286, kwiatkowski@chop.edu
Jaladhikumar Patel, 267-426-5602, patelj23@chop.edu
Locations
Children's Hospital of PhiladelphiaRECRUITING
Philadelphia, Pennsylvania
Contacts:
- Janet Kwiatkowski, MD, 215-590-5286, kwiatkowski@chop.edu
- Jaladhikumar Patel, 267-426-5602, patelj23@chop.edu
Sponsors
lead: Children's Hospital of Philadelphia
