Stem Cell Transplantation for Fanconi Anemia

Phase 2TerminatedNCT00167206

Masonic Cancer Center, University of Minnesota16 enrolled

Overview

The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.

All subjects will be given the same treatment regimen of total body irradiation (TBI), Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate donor stem cell transplant. Since this treatment regimen has been given before, without thymic shielding, we will compare the outcomes of these patients with the historical data from subjects who did not receive thymic shielding.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fanconi Anemia

Interventions

Hematopoietic Stem Cell TransplantPROCEDURE

Bone marrow failure may be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover.

Thymic Shielding During RadiationPROCEDURE

protecting the thymus during total body radiation (450 cGy administered)

Total Body IrradiationPROCEDURE

Eligibility

Sex: ALLMin age: 1 DayMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients must be less than (\<) 18 years of age with a diagnosis of Fanconi anemia. * Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal to (≤)1 antigen mismatched related (non-HLA-matched sibling) or \<1 antigen mismatched unrelated UCB donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. * Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below. * Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions * Platelet count \<20 x 10\^9/L * ANC \<5 x 10\^8/L * Hgb \<8 g/dL * Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies * High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations) * Adequate major organ function including * Cardiac: ejection fraction greater than (\>)45% * Hepatic: bilirubin, AST/ALT, ALP \<2 x normal * Karnofsky performance status \>70% or Lansky performance status \>50% * Women of child-bearing age must be using adequate birth control and have a negative pregnancy test Exclusion Criteria: * Available HLA-genotypically identical related donor * History of gram negative sepsis or systemic fungal infection (proven or suspected based on radiographic studies) * Refractory anemia with excess blasts, or leukemia * Active central nervous system (CNS) leukemia at time of hematopoietic cell transplant (HCT) * History of squamous cell carcinoma of the head/neck/cervix within 2 years of HCT * Pregnant or lactating female * Prior radiation therapy preventing use of total body irradiation (TBI) 450 centigray (cGy)

Locations (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment

Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10\^9/Liter.

Time frame: Day 42 after hematopoietic cell transplant

Secondary Outcomes

Number of Patients Who Exhibited Secondary Graft Failure

Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells.

Time frame: Day 100 after hematopoietic cell transplant

Number of Patients With Acute Graft Versus-Host Disease (aGVHD)

Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.

Time frame: Day 100 after hematopoietic cell transplant

Number of Patients With Chronic Graft Versus-Host Disease (GVHD)

Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.

Time frame: 1 year after hematopoietic cell transplant

Number of Patients Who Exhibited Regimen-related Toxicity (RRT)

Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given.

Data from ClinicalTrials.gov

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