Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

• Status: Completed
• Trial end date: 2020-10-10
• Target enrollment: 0
• Participant gender: All

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 transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia.

PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Antilymphocyte Serum; Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Lenograstim; Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Vidarabine

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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 Exclusion Criteria:

• Donor is a lactating female.