Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed By Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia - NCT00630253-55455(Clinical Trial 207086)
ClinicalConnection.com has recently undergone an update and this page may no longer be up-to-date. Please Search For Clinical Trials to view the most current clinical trials listings.
| City: |
|
Minneapolis |
|
State:
|
|
MN |
| Zip Code: |
|
55455 |
| Conditions: |
|
Fanconi Anemia |
| Purpose: |
|
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor
stem cell transplant helps stop the growth of abnormal cells. It also helps stop the
patient's immune system from rejecting the donor's stem cells. When the healthy stem cells
from a donor are infused into the patient, they may help the patient's bone marrow make stem
cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells
can make an immune response against the body's normal cells. Giving antithymocyte globulin
and removing the T cells from the donor cells before transplant and giving cyclosporine
before and after transplant may stop this from happening.
PURPOSE: This phase I/II trial is studying the side effects of cyclophosphamide,
fludarabine, and antithymocyte globulin followed by donor stem cell transplant and to see
how well it works in treating patients with Fanconi anemia.
|
| Study summary: |
|
OBJECTIVES:
Primary
- To determine the probability of engraftment in patients with Fanconi anemia treated
with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by
HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that
is T-cell depleted.
Secondary
- To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in
patients treated with this regimen.
- To evaluate the incidence of regimen-related toxicity in these patients.
- To evaluate the 1-year survival of patients treated with this regimen.
- To evaluate the incidence of late secondary malignancies (e.g., squamous cell carcinoma
of the head and neck or cervix) in patients treated with this regimen.
OUTLINE:
- Preparative cytoreductive therapy: Patients receive cyclophosphamide IV over 2 hours on
days -6 to -3 and fludarabine phosphate IV over 30 minutes and anti-thymocyte globulin
IV over 4-6 hours on days -6 to -2.
- T-cell depleted donor hematopoietic stem cell transplantation: Patients undergo T-cell
depleted donor bone marrow or umbilical cord blood stem cell transplantation on day 0.
Patients also receive filgrastim (G-CSF) IV beginning on day 1 and continuing until
blood counts recover.
- Graft-versus-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours or
orally every 8-12 hours beginning on day -3 and continuing until day 100, followed by a
taper. Patients will receive Mycophenolate Mofetil (MMF) therapy beginning on day -3
through day +30 or for 7 days after engraftment, whichever day is later, if no acute
GVHD. Engraftment is defined as 1st day of 3 consecutive days of absolute neutrophil
count [ANC] > 0.5 x 10^9/L.
After completion of study therapy, patients are followed periodically. |
| Criteria: |
|
Inclusion Criteria:
- Patients must be <60 years of age with a diagnosis of Fanconi Anemia (FA).
- Patients must have an HLA-A, B, DRB1 identical sibling 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 moderately severe aplastic anemia (AA), early
myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal
abnormalities.
- In patients <18 years of age, moderately severe aplastic anemia is defined as
having at least one of the following:
- platelet count <40 x 10^9/L
- absolute neutrophil count (ANC) <10 x 10^8/L
- Hgb <9 g/dL
- In patients 18-60 years of age, moderately severe aplastic anemia is defined as
having at least one of the following:
- platelet count <20 x 10^9/L
- absolute neutrophil count ANC <5 x 10^8/L
- Hgb <8 g/dL
- Early myelodysplastic syndrome, with multilineage dysplasia with < 5% blasts,
with or without chromosomal anomalies.
- Adequate major organ function including:
- Cardiac: ejection fraction >45%
- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- Karnofsky performance status >70% or Lansky >50%
- Women of child bearing age must be using adequate birth control and have a negative
pregnancy test.
Exclusion Criteria:
- Active bacterial infection within one week of hematopoietic cell transplant (HCT)
- Active fungal infection at time of HCT.
- Late MDS with greater than 5% blasts in bone marrow.
- Acute myelogenous leukemia (AML) or history of AML
- Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2
years of HCT.
- Pregnant or lactating female. |
|
|
|
| Study is available at: |
|
Masonic Cancer Center, University of Minnesota
Minneapolis, MN 55455
United States
Primary Contact:
Timothy Krepski
Email: tkrepsk1@fairview.org
Phone: 612-273-2800
Secondary Contact:
Margaret MacMillan, M.D.
Email: macmi002@umn.edu
Phone: 612-626-2778 |
|
|
If you are interested in this clinical trial please use the contact information above. If you would like to get additional information about this clinical trial please visit ClinicalTrials.gov.
|
| Trials Alerts: |
|
If you would like to be
notified of new clinical trials as they become available please
register for a free account.
|
|
| Data Source: |
|
ClinicalTrials.gov |
| Date Processed: |
|
March 22, 2011 |
Modifications to
this listing: |
|
Only selected fields are shown, please use the link
above to view all information about this clinical trial. |
|
|
|
|
|
|
|
|