What will happen in the study?
You, as a participant or as a parent/guardian, will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.
What is involved:
- Screening procedures will be done to determine if you are eligible to participate in this study.
- A surgical procedure, called a bone marrow harvest, will be performed to collect blood making cells.
- A chemotherapy drug called melphalan will be given to get you ready to receive the “gene transfer” blood-making cells.
- The “gene transfer” blood-making cells that were collected from your bone marrow harvest will be given back to you via a vein in your arm.
- You will be followed for approximately 15 years.