Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT00258427

Hematopoietic Stem Cell Transplantation in High Risk Patients With 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

  • Determine the tolerability of mycophenolate mofetil in these patients.
  • Determine the incidence of acute and chronic graft-vs-host disease in patients treated with this regimen.
  • Determine the incidence of major infections in patients with a history of major infections treated with this regimen.
  • Determine the incidence of relapse in patients with refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute myeloid leukemia treated with this regimen
  • Determine the probability of 1-year survival of patients treated with this regimen.

OUTLINE: Patients are stratified according to donor/recipient HLA type (identical vs other).

  • Cytoreductive combination chemotherapy: Patients receive busulfan intravenously (IV) over 2 hours twice daily on days -7 and -6 and cyclophosphamide IV over 2 hours and fludarabine IV over 30 minutes once daily on days -5 to -2.
  • Graft failure prophylaxis: Patients receive methylprednisolone IV twice daily on days -5 to 30 and anti-thymocyte globulin IV over 4-6 hours twice daily on days -5 to -1.
  • Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours twice daily on days -3 to 100 (if patient has a matched sibling donor) or days -3 to 180 (if patient has another donor type). Patients also receive mycophenolate mofetil orally or IV twice daily on days -3 to 45.
  • Allogeneic hematopoietic stem cell transplantation (HSCT): Patients undergo allogeneic HSCT (using bone marrow or umbilical cord blood) on day 0. Patients receive filgrastim (G-CSF) subcutaneously beginning on day 1 and continuing until blood counts recover.

After completion of study treatment, patients are followed periodically for 3 years.

Status: 
Recruiting
Study Date: 
Fri, 11/05/2010 to Sun, 01/01/2017
Associated Drug(s): 
Intervention: 
Biological: anti-thymocyte globulin Given 15 mg/kg/day intravenously every 12 hours on Days -5 through -1. Other Name: ATG Biological: filgrastim given 5 mcg/kg/day intravenously on Day 1 (continue until absolute neutrophil count (ANC) ≥2.5 x 10^9/L) Other Name: G-CSF Drug: busulfan Busulfan 0.8 mg/kg intravenously (IV) every 12 hours on Days -7 and -6 (1.0 mg/kg IV if <4 years old) Other Name: Busulfex Drug: cyclophosphamide 10 mg/kg intravenously (IV) on Days -5 through -2. Other Name: Cytoxan Drug: fludarabine phosphate 35 mg/m^2 intravenously (IV) on Days -5 through -2. Other Name: Fludara Drug: methylprednisolone 1 mg/kg intravenously (IV) every 12 hours on Days -5 through -1. Other Name: Medrol Biological: Hematopoietic stem cell transplantation Infused on Day 0 - Donor bone marrow or umbilical cord blood will be collected in the usual sterile manner using established parameters determined by the National Marrow Donor Program. Other Name: HSCT