RecruitingPhase 2NCT02143830

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Sponsor

Children's Hospital Medical Center, Cincinnati

Enrollment

70 participants

Start Date

Apr 1, 2014

Study Type

INTERVENTIONAL

Conditions

Fanconi Anemia Severe Marrow Failure Myelodysplastic Syndrome (MDS)Acute Myelogenous Leukemia (AML)

Summary

The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.

Eligibility

Min Age: 3 Months

Plain Language Summary

Simplified for easier understanding

This trial is testing a modified stem cell transplant approach using carefully adjusted chemotherapy (without radiation) for people with Fanconi anemia — a rare inherited condition that impairs the body's ability to repair DNA — who have developed serious blood problems like bone marrow failure, pre-leukemia, or leukemia. **You may be eligible if...** - You have a confirmed diagnosis of Fanconi anemia - You have developed severe aplastic anemia (bone marrow failure), myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML) - A suitable matched unrelated or related donor (not a fully matched sibling) has been identified - Your heart, liver, kidney, and lung function are adequate - Your overall health and activity level are reasonable (Karnofsky/Lansky score above 70%) **You may NOT be eligible if...** - Your physical condition or organ function does not meet the minimum requirements - You do not have a suitable donor Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGBusulfan

A standard dose of busulfan, associated with excellent outcomes in our previous trial will be used for young patients with marrow aplasia (arm A). A higher dose of busulfan will be used in younger patients with MDS and AML (arm B) to maximize disease control. A lower dose of busulfan will be used in older patients (arm C) to minimize toxicity.

DRUGCyclophosphamide

Arms A, B and C - Cytoxan will be given as a 1-2 hour infusion for 4 days. The dose will be adjusted according to patients ideal body weight for obese patients.

DRUGFludarabine

Arms A, B and C - Fludarabine will be given IV over 30 minutes daily for 4 days. The dose will be adjusted according to renal function according to Institutional guidelines.

DRUGrabbit ATG

Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment.

DRUGG-CSF

All patients will also receive G-CSF post-transplant to foster engraftment.

BIOLOGICALPeripheral blood stem cell

The source of stem cells for all patients will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 4-6 days. T-cell depletion will be uniformly performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device).