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HSCT for Fanconi Anemia with Risk-Adjusted Chemotherapy

This study is testing a new way to prepare patients with Fanconi Anemia for a stem cell transplant. It uses lower doses of certain chemotherapy drugs like Busulfan, Cyclophosphamide, and Fludarabine, along with rabbit ATG (a drug to prevent rejection) and G-CSF (to help new cells grow). The goal is to reduce side effects while still effectively treating severe bone marrow failure, myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML). You might be able to join if you have Fanconi Anemia and one of these blood conditions. The researchers will be looking to see if the transplant is successful and if your body accepts the new cells, measured over 5 years. The study is currently unclear on its recruitment status, but plans to enroll 70 patients.

Study design
This is a Phase II interventional study with three arms, designed to enroll 70 participants. It is testing different doses of Busulfan based on your specific condition.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will measure graft failure or rejection for 5 years after the transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT02143830

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Recruiting
PHASE2Ages 3+InterventionalTreatment
Children's Hospital Medical Center, Cincinnati
~70 participants
Updated 2025-11-12 on ClinicalTrials.gov
What's tested:BusulfanCyclophosphamideFludarabinerabbit ATGG-CSFPeripheral blood stem cell

At a glance

Recruiting sites
2 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Graft Failure or Rejection
Measured over 5 years

Conditions

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

Explore related trials

Acute Myeloid LeukemiaFanconi AnemiaMyelodysplastic SyndromeSevere Aplastic Anemia

Where it's being run

3 sites across 3 states
New York1
Ohio1
Washington1

Study leadership

  • Parinda Mehta, MD · PRINCIPAL_INVESTIGATOR · CCHMC

Who to contact

Jamie Wilhelm
[email protected]
(513)803-1102
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients must have a diagnosis of Fanconi anemia
Patients must have one of the following hematologic diagnoses:
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:
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

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

What this trial measures

  • Graft Failure or Rejection5 years

    Primary non-engraftment is diagnosed when the patient fails to achieve an ANC \>=500/mm3 at any time in the first 28 days post-transplant. If (1) after achievement of an absolute neutrophil count (ANC) \>=500/mm3, the ANC declines to \<500/mm3 for more than 3 consecutive days in the absence of relapse, or, (2) there is absence of donor cells in the marrow and/or blood as demonstrated by chimerism assay in the absence of relapse, a diagnosis of secondary graft failure is made. The patient is not evaluable for graft failure or rejection if recurrence of host MDS is detected concurrently.