Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00258427|
Recruitment Status : Recruiting
First Posted : November 24, 2005
Last Update Posted : February 12, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|Fanconi Anemia||Biological: anti-thymocyte globulin Biological: filgrastim Drug: busulfan Drug: cyclophosphamide Drug: fludarabine phosphate Drug: methylprednisolone Biological: Hematopoietic stem cell transplantation||Phase 2|
- 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.
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02|
|Actual Study Start Date :||March 26, 2002|
|Estimated Primary Completion Date :||January 2020|
|Estimated Study Completion Date :||January 2021|
Experimental: transplant in fanconi anemia patients
Cytoreductive preparative regimen consisting of busulfan, cyclophosphamide, fludarabine phosphate, methylprednisolone, and antithymocyte globulin (ATG) followed by hematopoietic stem cell transplantation (HSCT) and post-transplant use of bone marrow-stimulating filgrastim.
Biological: anti-thymocyte globulin
Given 15 mg/kg/day intravenously every 12 hours on Days -5 through -1.
Other Name: ATG
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
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
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
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
- Percent of Graft Failure [ Time Frame: Day 30 ]Graft failure = ANC <5 x 10^8/L by day 30.
- Incidence of Acute and Chronic Graft-Versus-Host Disease [ Time Frame: Day 42 and 1 Year ]
- Incidence of Relapse [ Time Frame: 1 Year ]
- Incidence of Major Infections [ Time Frame: Day 1 through End of Treatment ]
- Transplant-Related Toxicity [ Time Frame: Day 100 ]
- Overall Survival [ Time Frame: 1 Year ]cumulative proportion surviving
- Incidence of Chronic Graft-Versus-Host Disease [ Time Frame: Day 42 and 1 Year ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00258427
|Contact: Timothy Krepskiemail@example.com|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Timothy Krepski 612-273-2800 firstname.lastname@example.org|
|Principal Investigator: Margaret MacMillan, M.D.|
|Principal Investigator:||Margaret MacMillan, MD||Masonic Cancer Center, University of Minnesota|