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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-01-31

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Adult Acute Myeloid Leukemia in Remission Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Childhood Acute Myeloid Leukemia in Remission Childhood Myelodysplastic Syndromes Fanconi Anemia Previously Treated Myelodysplastic Syndromes

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment (allogeneic bone marrow or PBSC transplantation)

NON-MYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate 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 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.

Group Type EXPERIMENTAL

fludarabine phosphate

Intervention Type DRUG

Given IV

cyclosporine

Intervention Type DRUG

Given IV or PO

total-body irradiation

Intervention Type RADIATION

Undergo TBI

allogeneic bone marrow transplantation

Intervention Type PROCEDURE

Undergo allogeneic bone marrow transplantation

allogeneic hematopoietic stem cell transplantation

Intervention Type PROCEDURE

Undergo allogeneic PBSC transplantation

peripheral blood stem cell transplantation

Intervention Type PROCEDURE

Undergo allogeneic PBSC transplantation

mycophenolate mofetil

Intervention Type DRUG

Given PO or IV

Interventions

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fludarabine phosphate

Given IV

Intervention Type DRUG

cyclosporine

Given IV or PO

Intervention Type DRUG

total-body irradiation

Undergo TBI

Intervention Type RADIATION

allogeneic bone marrow transplantation

Undergo allogeneic bone marrow transplantation

Intervention Type PROCEDURE

allogeneic hematopoietic stem cell transplantation

Undergo allogeneic PBSC transplantation

Intervention Type PROCEDURE

peripheral blood stem cell transplantation

Undergo allogeneic PBSC transplantation

Intervention Type PROCEDURE

mycophenolate mofetil

Given PO or IV

Intervention Type DRUG

Other Intervention Names

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2-F-ara-AMP Beneflur Fludara ciclosporin cyclosporin cyclosporin A CYSP Sandimmune TBI bone marrow therapy, allogeneic bone marrow therapy, allogenic transplantation, allogeneic bone marrow transplantation, allogenic bone marrow PBPC transplantation PBSC transplantation peripheral blood progenitor cell transplantation transplantation, peripheral blood stem cell Cellcept MMF

Eligibility Criteria

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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

Eligible Sex

ALL

Accepts Healthy Volunteers

No