Status:
COMPLETED
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Fanconi Anemia
Eligibility:
All Genders
Up to 44 years
Phase:
PHASE2
Brief Summary
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell tra...
Detailed Description
OBJECTIVES: Primary * Determine whether the incidence of neutrophil engraftment is acceptable in high-risk patients with Fanconi's anemia treated with busulfan, cyclophosphamide, fludarabine, and an...
Eligibility Criteria
Inclusion
- Patients must be \<45 years of age with a diagnosis of Fanconi anemia with:
- Biallelic BRCA2 mutations, or
- Aplastic anemia, or advanced myelodysplastic syndrome (MDS) (MDS with ≥5% blasts), or acute leukemia who are ineligible for total body irradiation. Aplastic anemia is defined as having at least one of the following (with or without cytogenetic abnormalities): platelet count \<20 \* 10\^9, - absolute neutrophil count (ANC) \<5 \* 10\^8/L, - Hgb \<8 g/dL
- Patients must have an HLA-A, B, DRB1 identical or 1 antigen mismatched related or unrelated BM donor or have an HLA-A, B, DRB1 identical, 1 antigen or 2 antigen mismatched related or unrelated umbilical cord blood (UCB) donor. Patients and donors will be typed for HLA-A and B using serological level typing and for DRB1 using high resolution molecular typing.
- Adequate major organ function including:
- Cardiac: ejection fraction \>45%
- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites, no cirrhosis)
- Karnofsky performance status \>70% or Lansky \>50%
- Women of child bearing potential must be using adequate birth control and have a negative pregnancy test.
Exclusion
- Active CNS leukemia at time of HSCT.
- Active uncontrolled infection within one week of hematopoietic stem cell transplant (HSCT).
- Pregnant or lactating female.
- Donor Inclusion Criteria:
- Donor must be in good health based on review of systems and results of physical examination.
- Donor must have a normal hemoglobin, white count, platelet count and partial thromboplastin time (PTT), and a negative diepoxybutane (DEB) test.
- HIV-NAT negative, HTLV-1, HTLV-2 negative, Hepatitis B and C negative.
- Female donors of childbearing potential must have a negative pregnancy test.
- Unrelated donors must agree to peripheral blood stem cell (PBSC) donation
- Donor
Key Trial Info
Start Date :
March 26 2002
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 10 2020
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT00258427
Start Date
March 26 2002
End Date
October 10 2020
Last Update
December 2 2021
Active Locations (1)
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1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455