Clinical Trials Logo

Clinical Trial Summary

RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant and giving cyclosporine before and after transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the side effects of cyclophosphamide, fludarabine, and antithymocyte globulin followed by donor stem cell transplant and to see how well it works in treating patients with Fanconi anemia.


Clinical Trial Description

OBJECTIVES: Primary - To determine the probability of engraftment in patients with Fanconi anemia treated with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted. 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT00630253
Study type Interventional
Source Masonic Cancer Center, University of Minnesota
Contact
Status Completed
Phase Phase 1/Phase 2
Start date February 17, 2000
Completion date October 10, 2020

See also
  Status Clinical Trial Phase
Completed NCT02931071 - Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1 Phase 2
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Recruiting NCT00084695 - Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases Phase 2
Completed NCT00000603 - Cord Blood Stem Cell Transplantation Study (COBLT) Phase 2
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT01146210 - Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia N/A
Completed NCT01082133 - Multicenter Transplant Study for Fanconi Anemia Phase 2
Completed NCT00965666 - Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia Early Phase 1
Terminated NCT00290628 - Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer N/A
Recruiting NCT00027274 - Cancer in Inherited Bone Marrow Failure Syndromes
Terminated NCT01001598 - Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita Phase 1/Phase 2
Recruiting NCT03206086 - Eltrombopag for People With Fanconi Anemia Phase 2
Recruiting NCT03579875 - Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Phase 2
Recruiting NCT05598515 - Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia N/A
Active, not recruiting NCT03476330 - Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia Phase 2
Terminated NCT05910853 - Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
Completed NCT03609840 - Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Completed NCT00479115 - Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 Phase 1/Phase 2
Completed NCT00352976 - TBI Dose De-escalation for Fanconi Anemia Phase 2/Phase 3
Terminated NCT03600909 - A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia Phase 2