Dose Finding Study of Thymoglobulin (ATG) in Patients With Steroid-Refractory Acute Graft Versus Host Disease (aGVHD)
GvHD is a common side effect of a stem cell transplant. It is caused by a type of white
blood cell (a lymphocyte). ATG is designed to kill lymphocytes and is commonly used to help
prevent or treat GvHD and to treat steroid-refractory aGvHD.
Before you can start treatment on this study, you will have "screening tests." These tests
will help the doctor decide if you are eligible to take part in this study. You will be
asked questions about your medical history. You will have a complete physical exam. You
will have blood drawn (about 2 tablespoons) for routine tests and to check for any viral
infections. Women who are able to have children must have a negative blood (about 1
teaspoon) or urine pregnancy test. You will also have a biopsy of the site that is suspected
to be affected by aGvHD. The study doctor will describe the type of biopsy procedure you
will have, which will depend on the part of the body that is affected by the disease. If
you had an earlier biopsy performed that confirmed aGvHD, you will not need a biopsy
repeated for this study.
If you are found to be eligible to participate in this study, you will be randomly assigned
(as in the toss of a coin) to 1 of 2 groups. Participants in Group 1 will receive a higher
dose of ATG by vein. Participants in Group 2 will receive a lower dose of ATG by vein. You
will have an equal chance of being in 1 of the 2 groups. Participants in both groups will
receive doses of ATG over 4-6 hours, 1 time a day every other day for a total of 3 doses.
During this study, you will be able to remain on steroids and any other drugs you may be
receiving for treatment of aGvHD (such as tacrolimus or cyclosporine). You will also have
blood drawn (about 1 teaspoon) to learn the effect of ATG on the lymphocytes.
You will be taken off this study, if the disease is stable as determined by the study
doctor, if the disease gets worse, or you experience any intolerable side effects. If you
are taken off this study, your study doctor will discuss other treatment options with you.
After you finish receiving the study medication, you will come to the clinic at regularly
scheduled follow-up visits. At first, you will be seen in the clinic once a week for 1
month. You will also have additional blood drawn (about 2 teaspoons) for biomarker testing
(testing that looks at how the drug is working in your body). After the first month of
follow-up visits, you will come in for visits every other week while you are on this study,
which will continue until you have at least 2 consecutive (back to back) evaluations of the
disease status to see if the disease is stable.
This is an investigational study. ATG is FDA approved and commercially available. You
and/or your insurance provider will be financially responsible for the cost of ATG while you
are on this study. Up to 40 patients will take part in this study. All will be enrolled at
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Response assessed at day 56, and a complete response or partial response considered a success. A toxic event defined as a life threatening infection, any death due to infection, or any death considered to be directly related to the administration of ATG.
Amin M. Alousi, MD
M.D. Anderson Cancer Center
United States: Institutional Review Board
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