Clinical Trial Detail

This is an unrelated donor transplantation study for patients up to age 65 with severe aplastic anemia.The purpose of this study is to determine what dose of cyclophosphamide should be used, along with other medications before transplantation, to improve the safety and effectiveness of transplantation.

The chemotherapy and irradiation given just before a transplant is called the conditioning regimen. Participants in this study will receive a conditioning regimen consisting of fludarabine, cyclophosphamide, and antithymocyte globulin (ATG), as well as low-dose total body irradiation.

Different groups of participants will receive different amounts of cyclophosphamide, of which they will be informed.

Study participants will then receive a bone marrow transplant using cells from an unrelated donor.

Most of the care will be given at the Seattle Cancer Care Alliance (SCCA) outpatient clinic. During the transplant and the initial recovery period, and if needed at different time points after the transplant, participants will be admitted to the UW Medical Center hospital.

Participants will be asked to come to the SCCA clinic on a regular basis for exams and blood tests for about 3 months after the transplant. Participants will be followed by their personal physicians for up to 2 years.

1. Patient is 65 years of age or younger.

2. Patient has a diagnosis of Severe Aplastic Anemia.

3. Patient has a suitable, unrelated donor. (The donor cannot be a family member.)

4. Patient may be either female or male.

Other eligibility criteria may apply.

1. Clonal cytogenetic abnormalities associated with MDS or AML on marrow examination.

2. Diagnosis of Fanconi anemia.

3. Diagnosis of other “congenital” marrow failure states (such as Diamond-Blackfan, Shwachmann-Diamond, congenital amegakaryocytosis).

4. Symptomatic or uncontrolled heart failure or coronary artery disease.

5. Uncontrolled infection.

6. Blood test results show presence of HIV infection.

7. Pregnancy or breastfeeding.

8. Known allergy to ATG, cyclosporine or tacrolimus.

9. Patient is also enrolled in another phase I study.

10. Patient has had a prior allogeneic marrow or stem cell transplant.

11. Patient has had a prior malignancy, except resected basal cell carcinoma or treated carcinoma in-situ.

Other exclusion criteria may apply.