Clinical Trial Goal
To find out if the SUN regimen before allogeneic BMT with a matched related donor is safe and works well to treat SCD in children, teens and young adults
You may be able to join this trial if you:
Your child:
- Is 2 – 25 years old
- Has SCD that doctors consider severe
- Has a brother of sister who is a full match to them and is able to be their donor
You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
Alemtuzumab is a monoclonal antibody that targets CD52 on certain cells.
Sirolimus is a drug that slows down the immune system.
Before transplant, your child will get the SUN regimen to prepare their body for BMT. This includes:
Sirolimus is a drug that slows down the immune system.
Before transplant, your child will get the SUN regimen to prepare their body for BMT. This includes:
- Alemtuzumab – Given as intravenous (IV) infusions 5 times during the week before BMT
- Sirolimus – Given as a pill your child takes by mouth 1 time the day before BMT
- Total Body Irradiation (TBI) – A treatment done 1 time 2 days before BMT to prepare your child’s body for transplant. In this trial, your child will get low dose TBI
On transplant day, the blood-forming cells from your child’s donor are given to them through an IV infusion.
After transplant, your child will get treatment to help prevent graft-versus-host-disease (GVHD):
- Sirolimus – A pill that your child takes by mouth 1 time each day starting on the day of BMT
Your child may continue treatment with sirolimus for up to 1 year. After BMT, the clinical trial doctors will check your child’s health for about 3 months.
The Food and Drug Administration (FDA) has approved the drugs used in this trial. Using the SUN regimen before allogeneic BMT to treat SCD in children, teens and young adults is new and unproven.
Contacts
Robert Nickel, MD, 202-476-5000, RNickel@childrensnational.org
Fahmida Hoq, MBBS, 202-476-5000, fhoq@childrensnational.org
Locations
Sponsors
lead: Robert Nickel, collaborator: Alberta Children's Hospital, collaborator: The Hospital for Sick Children, collaborator: Levine Children's Hospital, collaborator: Ann & Robert H Lurie Children's Hospital of Chicago, collaborator: Nationwide Children's Hospital, collaborator: The Children's Hospital at Montefiore, collaborator: Columbia University
