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RECRUITING

NCT02143830

PHASE2

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Sponsor: Children's Hospital Medical Center, Cincinnati

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

3 Months - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2014-04

Completion Date

2028-12

Last Updated

2025-11-12

Healthy Volunteers

Conditions

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

Interventions

DRUG

Busulfan

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.

DRUG

Cyclophosphamide

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.

DRUG

Fludarabine

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.

DRUG

rabbit ATG

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

DRUG

G-CSF

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

BIOLOGICAL

Peripheral 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).

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

Locations (3)

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Clinical Research Directory

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (3)