NCT00258427

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 44 Years
• Healthy volunteers: Not accepted

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.

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 antithymocyte globulin followed by allogeneic hematopoietic stem cell transplantation. 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.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
BIOLOGICAL	anti-thymocyte globulin	Given 15 mg/kg/day intravenously every 12 hours on Days -5 through -1.
BIOLOGICAL	filgrastim	given 5 mcg/kg/day intravenously on Day 1 (continue until absolute neutrophil count (ANC) ≥2.5 x 10\^9/L)
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)
DRUG	cyclophosphamide	10 mg/kg intravenously (IV) on Days -5 through -2.
DRUG	fludarabine phosphate	35 mg/m\^2 intravenously (IV) on Days -5 through -2.
DRUG	methylprednisolone	1 mg/kg intravenously (IV) every 12 hours on Days -5 through -1.
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.

Timeline

• Start date: 2002-03-26
• Primary completion: 2020-10-10
• Completion: 2020-10-10
• First posted: 2005-11-24
• Last updated: 2021-12-02
• Results posted: 2021-12-02

Locations

1 site across 1 country: United States