Clinical Trial Goal
To find out if emapalumab is safe and works well to treat SAA that has not yet been treated in children and young adults
You may be able to join this trial if you:
Your child:
- Is 0 - 25 years old
- Has SAA that has not yet been treated
- Does not have any of the following:
- Leukemia
- Myelodysplastic syndrome (MDS)
- You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
Anti-thymocyte globulin (ATG) and cyclosporine are standard drugs used to prevent graft-versus-host disease (GVHD) or to slow down the immune system.
Emapalumab is a monoclonal antibody that targets interferon-y in certain cells.
In this trial, your child will be randomized to 1 of 2 groups. Once randomized, your child will be told what group you/they're in:
Emapalumab is a monoclonal antibody that targets interferon-y in certain cells.
In this trial, your child will be randomized to 1 of 2 groups. Once randomized, your child will be told what group you/they're in:
- Group 1 –Emapalumab plus BMT
- Group 2 – Emapalumab plus standard IST
Randomized means doctors will use a computer to assign your child to either group. A computer assigns by chance, like flipping a coin or drawing a name out of a hat. You, your child's doctor or the clinical trial doctor won’t have any control over which group is assigned. This means you won’t be able to choose your child's group.
In Group 1, your child will have a BMT
First, they will get:
- Emapalumab – Given as intravenous (IV) infusions. The dose your child will get depends on when they start the trial and how safe it has been
On transplant day, the blood-forming cells from your child or your child's donor are given through an IV infusion.
In Group 2, your child will get standard immunosuppressive therapy with:
- ATG - A pill taken by mouth. The dose your child will get depends on when they start the trial and how safe it has been
- Cyclosporine – Given as IV infusions. The dose your child will get depends on when they start the trial and how safe it has been
Then, they will get:
- Emapalumab – Given as IV infusions. The dose your child will get depends on when they start the trial and how safe it has been
Your child will have biopsies to see how well the treatment is working.
The Food and Drug Administration (FDA) has approved all of the drugs used in this trial. The FDA has approved emapalumab to treat hemophagocytic lymphohistiocytosis. Using emapalumab to treat SAA is new and unproven.
The Food and Drug Administration (FDA) has approved all of the drugs used in this trial. The FDA has approved emapalumab to treat hemophagocytic lymphohistiocytosis. Using emapalumab to treat SAA is new and unproven.
Contacts
Andromachi Scaradavou, MD, 1-833-MSK-KIDS, ScaradaA@mskcc.org
Jaap Jan Boelens, MD, PhD, 1-833-MSK-KIDS, boelensj@mskcc.org
Locations
Sponsors
lead: Memorial Sloan Kettering Cancer Center
