Clinical trial · Interventional
Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
Low-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow. It should also be less harmful (toxic). Researchers wish to test whether this approach can overcome the graft failure often seen when bone marrow or peripheral blood stem cells from an unrelated donor are used. Researchers also will look at whether the procedure is less toxic than a conventional bone marrow transplant (BMT).
Conditions
Conditions (11)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Adult Acute Myeloid Leukemia in Remission | Adult Acute Myeloid Leukemia | CURATED_BROADER | 0.78 |
| Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | — | UNRESOLVED | — |
| Adult Acute Myeloid Leukemia With Del(5q) | Acute Myeloid Leukemia with del(5q) | ONTOLOGY_EXACT | 0.85 |
| Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | — | UNRESOLVED | — |
| Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) | — | UNRESOLVED | — |
| Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia with t(16;16)(p13.1;q22); CBFB-MYH11 | ALIAS | 0.90 |
| Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Acute Myeloid Leukemia with t(8;21)(q22;q22.1); RUNX1-RUNX1T1 |
Interventions
Interventions (7)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| allogeneic hematopoietic stem cell transplantation | Procedure | — | UNRESOLVED |
| cyclosporine | Drug | — | UNRESOLVED |
| fludarabine phosphate | Drug | Fludarabine | ALIAS |
| mycophenolate mofetil | Drug | — | UNRESOLVED |
| peripheral blood stem cell transplantation | Procedure | — | UNRESOLVED |
| total-body irradiation | Radiation | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment (allogeneic bone marrow or PBSC transplantation)
- description
- NON-MYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate IV over 30 minutes on days -4 to -2, cyclosporine IV every 8-12 hours on days -3 to 0, and undergo low-dose TBI on day 0. TRANSPLANTATION: Patients undergo allogeneic bone marrow or PBSC transplantation on day 0. IMMUNOSUPPRESSION: Patients receive cyclosporine PO or IV every 8-12 hours on days 1-100 with taper to day 177, and mycophenolate mofetil PO or IV every 8 hours on days 0-40 with taper to day 96.
- interventionNames
- Drug: fludarabine phosphate
- Drug: cyclosporine
- Radiation: total-body irradiation
- Procedure: allogeneic bone marrow transplantation
- Procedure: allogeneic hematopoietic stem cell transplantation
- Procedure: peripheral blood stem cell transplantation
- Drug: mycophenolate mofetil
Primary outcomes (6)
- measure
- Engraftment, defined as donor chimerism (mixed or complete)
- timeFrame
- Day 28
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
Inclusion Criteria: * Any patient with marrow failure and increased chromosome fragility as determined in the diepoxybutane (DEB) or mitomycin C test * Any patient with Fanconi anemia (FA) with marrow failure meeting the following criteria: * Granulocyte count \< 0.2 x 10\^9/L * Platelet count \< 20 x 10\^9/L * Hemoglobin \< 8 g/dl * Corrected reticulocyte count \<1% * Any patient with FA as determined by DEB fragility, who has life-threatening marrow failure involving a single hematopoietic lineage * Any patient with FA and pre-existing cytogenetic abnormality including hematopoietic malignancy (myelodysplastic syndromes \[MDS\] or acute myeloid leukemia \[AML\]) in remission * DONOR: Unrelated Donors who are prospectively: Matched for human lymphocyte antigen (HLA)-DRB1 and DQB1 alleles (must be defined by high resolution typing); only a single allele disparity will be allowed for HLA -A, B, or C as defined by high resolution typing * DONOR: HLA typing will be performed at the highest level of resolution available at the time of transplant Exclusion Criteria: * Evidence for hematopoietic malignancy in relapse * Heart or lung disease that would prevent compliance with conditioning and GvHD regimen or would severely limit the probability of survival * Human immunodeficiency virus (HIV) seropositive patients * Females who are pregnant or breastfeeding, or unwilling to use contraceptive techniques during and for the 12 months following treatment * DONOR: Donors who by DEB testing are found to have FA * DONOR: Donors who test positive in the lymphocytotoxic crossmatch assay * DONOR: Donors who are HIV positive * DONOR: Donors who for other medical or psychological reasons are not suitable as donors
References
Publications (0)
Data not yet available