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HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

NCT02143830 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 70

Last updated 2025-11-12

No results posted yet for this study

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.

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).

Sponsors & Collaborators

Principal Investigators

  • Parinda Mehta, MD · CCHMC

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-04-30
Primary Completion
2026-12-31
Completion
2028-12-31

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02143830 on ClinicalTrials.gov