HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy



Status:Recruiting
Conditions:Blood Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Anemia, Anemia, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:Any
Updated:6/28/2018
Start Date:April 2014
End Date:May 2020
Contact:Jamie Wilhelm
Email:Jamie.Wilhelm@cchmc.org
Phone:(513)803-1102

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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

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.

The trial proposed is a three arm phase II treatment protocol designed to investigate the
safety and efficacy of risk-adjusted chemotherapy-based cytoreductive regimen plus a CD34+
selected T-cell depleted peripheral blood stem cell (PBSC) stem cell transplant for the
treatment of patients with Fanconi anemia and severe hematologic disease. Candidates for this
trial will include patients with Fanconi anemia presenting with severe marrow failure
(transfusion dependent) or myelodysplastic syndrome, or acute myelogenous leukemia for whom
an allogeneic stem cell transplant is indicated.

Inclusion Criteria:

- Patients must have a diagnosis of Fanconi anemia

- Patients must have one of the following hematologic diagnoses:

1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of <25% OR Severe
Isolated Single Lineage Cytopenia and at least one of the following features:

1. Platelet count <20 x 109/L or platelet transfusion dependence*

2. ANC <1000 x 109/L

3. Hgb <8 gm/dl or red cell transfusion dependence*

2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification

3. Acute Myelogenous Leukemia (untreated, in remission or with refractory or
relapsed disease)

- Donors will be either human leukocyte antigen (HLA) compatible unrelated or
HLA-genotypically matched related donors (no fully matched sibling donor).

- Patients and donors may be of either gender or any ethnic background.

- Patients must have a Karnofsky adult, or Lansky pediatric performance scale status >
70%.

- Patients must have adequate physical function measured by:

1. Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection
fraction (LVEF) at rest must be > 50% and must improve with exercise or 2)
Shortening Fraction > 29%

2. Hepatic: < 5 x upper limit of normal (ULN) alanine transaminase (ALT) and < 2.0
mg/dl total serum bilirubin.

3. Renal: serum creatinine <1.5 mg/dl or if serum creatinine is outside the normal
range, then CrCl > 50 ml/min/1.73 m2

4. Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted

- Each patient must be willing to participate as a research subject and must sign an
informed consent form.

- Female patients and donors must not be pregnant or breastfeeding at the time of
signing consent. Women must be willing to undergo a pregnancy test prior to transplant
and avoid becoming pregnant while on study.

Exclusion Criteria:

- Active CNS leukemia

- Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.

- Active uncontrolled viral, bacterial or fungal infection

- Patient seropositive for HIV-I/II; HTLV -I/II
We found this trial at
3
sites
1275 York Ave
New York, New York 10021
(212) 639-2000
Principal Investigator: Farid Boulad, MD
Phone: 646-888-5718
Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering Cancer Center — the world's oldest and...
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New York, NY
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Parinda Mehta, MD
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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1100 Fairview Avenue North
Seattle, Washington 98109
(206) 667-5000
Principal Investigator: K. Scott Baker, MD
Phone: 206-667-4222
Fred Hutchinson Cancer Research Center At Fred Hutchinson Cancer Research Center, our interdisciplinary teams of...
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Seattle, WA
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