RAFA

A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

What's the purpose of the trial?

The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
Trial status

Accepting patients

Phase
Phase 2
Enrollment
70
Last Updated
1 month ago
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Participating Centers

There are 2 centers participating in this trial. Enter a location below to find the closest center.

Experimental Treatments

Learn more about the experimental treatments being evaluated in this clinical trial.

  • Allogeneic Stem Cell Transplant is a type of stem cell transplant that utilizes a donor's pre-collected stem cells to rescue the bone marrow from the toxic effects of very high dose chemotherapy.
  • Antithymocyte Globulin (ATG) is an antibody preparation made from rabbits or horses immune cells altered with human thymocytes. ATG is used to prevent or treat acute cellular rejection after solid organ transplantation and as a therapy for certain other blood disorders.
  • Busulfan is a chemotherapy cancer medication that interferes with the growth and spread of cancer cells in the body, and may be used in the treatment of many different cancers.
  • Cyclophosphamide is a chemotherapy agent used in the treatment of cancer.
  • Fludarabine is a chemotherapy medication that may be used in the treatment of many different cancers.
  • Granulocyte Colony-Stimulating Factor is a glycoprotein used to stimulate the bone marrow to help with granulocyte and stem cell production.

Arms / Cohorts

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Accepting patients

Good Risk Patients

Accepting patients

Intermediate Risk Patients

Accepting patients

High Risk Patients

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