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aplastic anemia

Horse ATG in Patients With AA or Low/Int-1 Risk MDS

Study Drug Administration:

If you are found to be eligible to take part in this study, you will be admitted to the hospital. On the first day, before starting the hATG, a small amount of diluted hATG will be injected under your skin to make sure that you are not allergic to it. If you have a reaction, more testing may be done. If the reaction is severe, you will be taken off study.

On Days 1-4 you will receive methylprednisolone by vein over about 10 minutes. You will receive hATG by vein over 8 hours.

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerou

PRIMARY OBJECTIVES:

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

This will be an open-label, single arm, phase IIa study in subjects with AA or Hypoplastic MDS.

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results.