Phase 2
N=6
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia
Acute Myeloid Leukemia in Remission · de Novo Myelodysplastic Syndrome · Fanconi Anemia · Previously Treated Myelodysplastic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00453388 ↗Enrolled (actual)
6
Serious AEs
33.3%
Results posted
May 2017
Primary outcome: Primary: Number of Patients Who Engraft at Each Dose of TBI Used — 5; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Bone Marrow Transplantation (Procedure); Cyclophosphamide (Drug); Cyclosporine (Drug); Fludarabine Phosphate (Drug); Laboratory Biomarker Analysis (Other); Mycophenolate Mofetil (Drug); Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation (Procedure); Total-Body Irradiation (Radiation)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Fred Hutchinson Cancer Center
- Primary completion
- Jun 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Who Engraft at Each Dose of TBI Used |
5; 1 | — |
| PRIMARY Incidence of Grades III-IV Acute GVHD |
1; 0 | — |
| SECONDARY Incidence of Transplant-related Mortality |
1; 0 | — |
| SECONDARY Incidence of Adverse Events |
3; 0 | — |
Summary
This phase II trial studies how well total-body irradiation (TBI) works when given together with fludarabine phosphate and cyclophosphamide followed by donor bone marrow transplant, mycophenolate mofetil, and cyclosporine in treating patients with Fanconi anemia (FA). Giving low doses of chemotherapy, such as fludarabine phosphate and cyclophosphamide, and TBI before or after a donor bone marrow transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.
Eligibility Criteria
Inclusion Criteria
- Any patient with FA and bone marrow (BM) failure involving 2 of the following 3 lineages: granulocyte count = 5% as assessed by morphology
- Active infectious disease concerns
- Karnofsky performance score < 50 or Lansky performance score < 40
- DONOR: Donors found to have Fanconi anemia based on chromosomal breakage analysis
- DONOR: Donors who are not expected to meet the minimum target dose of marrow cells (1 x 10^8 nucleated cells/kg recipient ideal body weight [IBW]) or who are unwilling to be bone marrow donors
- DONOR: HIV-positive donors
- DONOR: Donors who are cross-match positive with recipient
- DONOR: Recipient homozygous at mismatched locus; if the recipient is homozygous at HLA-A, B, or C and the donor is mismatched at that locus, the donor should be avoided; exceptions must be discussed with the PI
Data sourced from ClinicalTrials.gov (NCT00453388). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.