Myelodysplastic Syndrome Clinical Trial

Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia

Summary

Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow. It should also be less harmful (toxic). Researchers wish to test whether this approach can overcome the graft failure often seen when bone marrow or peripheral blood stem cells from an unrelated donor are used. Researchers also will look at whether the procedure is less toxic than a conventional bone marrow transplant (BMT).

View Full Description

Full Description

PRIMARY OBJECTIVES:

I. To determine whether donor chimerism can be achieved in patients with Fanconi anemia receiving marrow or peripheral blood stem cell (PBSC) grafts from unrelated donors following low dose total body irradiation (TBI), fludarabine (fludarabine phosphate), mycophenolate mofetil, and cyclosporine.

II. To determine the lowest dose of TBI necessary to achieve donor chimerism in at least 80% of patients.

III. To determine the incidence of severe regimen-related toxicity.

SECONDARY OBJECTIVES:

I. To determine the survival of Fanconi anemia patients transplanted with unrelated donor marrow or PBSC grafts after conditioning with a non-myeloablative regimen.

II. To determine the incidence and severity of graft-vs-host disease (GVHD) incurred with unrelated bone marrow or PBSC grafts transplant patients with Fanconi anemia.

III. To determine if mixed chimerism results in amelioration of symptoms associated with bone marrow failure in patients with Fanconi anemia.

OUTLINE:

NON-MYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -4 to -2, cyclosporine IV every 8-12 hours on days -3 to 0, and undergo low-dose TBI on day 0.

TRANSPLANTATION: Patients undergo allogeneic bone marrow or PBSC transplantation on day 0.

IMMUNOSUPPRESSION: Patients receive cyclosporine orally (PO) or IV every 8-12 hours on days 1-100 with taper to day 177, and mycophenolate mofetil PO or IV every 8 hours on days 0-40 with taper to day 96.

After completion of study treatment, patients are followed up at 6 months and annually thereafter.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

Any patient with marrow failure and increased chromosome fragility as determined in the diepoxybutane (DEB) or mitomycin C test
Any patient with Fanconi anemia (FA) with marrow failure meeting the following criteria:
Granulocyte count < 0.2 x 10^9/L
Platelet count < 20 x 10^9/L
Hemoglobin < 8 g/dl
Corrected reticulocyte count <1%
Any patient with FA as determined by DEB fragility, who has life-threatening marrow failure involving a single hematopoietic lineage
Any patient with FA and pre-existing cytogenetic abnormality including hematopoietic malignancy (myelodysplastic syndromes [MDS] or acute myeloid leukemia [AML]) in remission
DONOR: Unrelated Donors who are prospectively: Matched for human lymphocyte antigen (HLA)-DRB1 and DQB1 alleles (must be defined by high resolution typing); only a single allele disparity will be allowed for HLA -A, B, or C as defined by high resolution typing
DONOR: HLA typing will be performed at the highest level of resolution available at the time of transplant

Exclusion Criteria:

Evidence for hematopoietic malignancy in relapse
Heart or lung disease that would prevent compliance with conditioning and GvHD regimen or would severely limit the probability of survival
Human immunodeficiency virus (HIV) seropositive patients
Females who are pregnant or breastfeeding, or unwilling to use contraceptive techniques during and for the 12 months following treatment
DONOR: Donors who by DEB testing are found to have FA
DONOR: Donors who test positive in the lymphocytotoxic crossmatch assay
DONOR: Donors who are HIV positive
DONOR: Donors who for other medical or psychological reasons are not suitable as donors

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 1

Estimated Enrollment:

2

Study ID:

NCT00093743

Recruitment Status:

Completed

Sponsor:

Fred Hutchinson Cancer Center

Check Your Eligibility

Let’s see if you might be eligible for this study.

What is your age and gender ?

Submit

There are 5 Locations for this study

See Locations Near You

Robert H. Lurie Comprehensive Cancer Center
Chicago Illinois, 60611, United States
Riley Hospital for Children
Indianapolis Indiana, 46202, United States
Vanderbilt-Ingram Cancer Center
Nashville Tennessee, 37232, United States
Huntsman Cancer Institute/University of Utah
Salt Lake City Utah, 84112, United States
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
Seattle Washington, 98109, United States

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 1

Estimated Enrollment:

2

Study ID:

NCT00093743

Recruitment Status:

Completed

Sponsor:


Fred Hutchinson Cancer Center

How clear is this clinincal trial information?

×

Introducing, the Journey Bar

Use this bar to access information about the steps in your cancer journey.

Please confirm you are a US based health care provider:

Yes, I am a health care Provider No, I am not a health care provider