Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT00630253

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

OBJECTIVES:

Primary

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.

Status: 
Recruiting
Study Date: 
Fri, 03/05/2010 to Wed, 06/01/2016
Intervention: 
Biological: anti-thymocyte globulin 30 mg/kg/day will be administered after MP on days -6, - 5, -4, -3 and -2. Other Name: ATGAM Drug: cyclophosphamide 5 mg/kg is to be given as a 2 hour infusion, Days -6 through -3. Other Name: Cytoxan Drug: fludarabine phosphate 35 mg/m^2 intravenously (IV) on days -6 through -2. Other Name: Fludara Procedure: hematopoietic stem cell transplantation Bone marrow or umbilical cord blood infusion on day 0. Other Name: stem cell transplant Drug: methylprednisolone MP 2 mg/kg/day intravenously every 24 hours will be given from day -6 until day +15 at which time it will be tapered 50% every 4 days to be discontinued by day +24 (i.e. MP 1 mg/kg/day on days +16-19, then 0.5 mg/kg/day on days +20-23, then discontinue). MP will be used as a premedication for ATG on day -6 to -2. Other Name: MP Drug: filgrastim Initiate G-CSF 5 mcg/kg per day intravenously (IV) continue until Absolute neutrophil count > or = 2.5 x 10^9/L Other Name: G-CSF Drug: cyclosporine cyclosporine IV over 2 hours or orally every 8-12 hours beginning on day -3 and continuing until day 100, followed by a taper Other Name: CSA Drug: Mycophenolate Mofetil 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. MMF will be given at a dose of 15 mg/kg/dose every 8 hours PO (to a maximum dose of 1 gram). Other Name: MMF