Phase 2
Completed N=31
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Source: ClinicalTrials.gov NCT00630253 ↗Enrolled (actual)
31
Serious AEs
3.2%
Results posted
Oct 2021
Primary outcomePrimary: Number of Participants Experiencing Graft Failure — 0; 0; 0 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Graft Failure |
0; 0; 0 | — |
| SECONDARY Number of Participants With Acute Graft-Versus-Host Disease (GVHD) |
0; 0; 0 | — |
| SECONDARY Number of Participants Experiencing Overall Survival |
15; 8; 5 | — |
| SECONDARY Number of Participants With Chronic Graft-Versus-Host Disease (GVHD) |
2; 0; 0 | — |
| SECONDARY Number of Participants With Transplant Related Deaths |
1; 0; 1 | — |
Eligibility Criteria
Inclusion Criteria
- Patients must be 45%
- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- Karnofsky performance status >70% or Lansky >50%
- Women of child bearing age must be using adequate birth control and have a negative pregnancy test.
Exclusion Criteria
- Active bacterial infection within one week of hematopoietic cell transplant (HCT)
- Active fungal infection at time of HCT.
- Late MDS with greater than 5% blasts in bone marrow.
- Acute myelogenous leukemia (AML) or history of AML
- Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2 years of HCT.
- Pregnant or lactating female.
Data sourced from ClinicalTrials.gov (NCT00630253). 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. Informational only — not medical advice.