Transplant for Patients with Fanconi Anemia Using Adjusted Chemotherapy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial aims to evaluate the safety and efficacy of a risk-adjusted chemotherapy regimen combined with a stem cell transplant for patients with Fanconi anemia and severe hematologic conditions. Participants will receive a preparative regimen that includes lower doses of busulfan, fludarabine, anti-thymocyte globulin, and cyclophosphamide, followed by a transplant using either mismatched related or matched unrelated donors. The study is designed to minimize transplant-related side effects while effectively treating severe marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia.

Why it matters

Who can join: the full rules

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

Where it's running

• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting
• Fred Hutchinson Cancer Research Center
  Seattle, Washington, United States Recruiting
• Memorial Sloan Kettering Cancer Center
  New York, New York, United States Completed

Study contacts

• Principal investigator: Parinda Mehta, MD — Cchmc
• Study coordinator: Jamie Wilhelm
• Email: Jamie.Wilhelm@cchmc.org
• Phone: (513)803-1102

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.