Study on Stem Cell Transplant for Patients with Fanconi Anemia Using Adjusted Chemotherapy

Sponsor: Children's Hospital Medical Center, Cincinnati

Sponsor score: 40
Inactive

ClinicalTrials ID:NCT02143830

Provider View

This clinical trial is exploring whether using lower doses of certain chemotherapy drugs can reduce side effects for patients with Fanconi Anemia undergoing a stem cell transplant. The study involves using donors who are either partially matched relatives or unrelated but matched donors.

Patient Parameters

Program Overview

The aim of this study is to find out if using lower doses of the chemotherapy drug busulfan and not using cyclosporine can lessen the side effects of stem cell transplants in patients with Fanconi Anemia. Patients will receive a transplant from either a partially matched relative or a matched unrelated donor. Before the transplant, they will undergo a treatment plan that includes busulfan, fludarabine, anti-thymocyte globulin, and cyclophosphamide.

Description

This study is a Phase II trial with three different treatment groups. It is designed to test the safety and effectiveness of a chemotherapy-based treatment plan that is adjusted based on the patient's risk level. This plan is combined with a special type of stem cell transplant for patients with Fanconi Anemia who have severe blood-related health issues. Eligible patients include those with severe bone marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia, who need a stem cell transplant from a donor.

Eligibility Criteria

Inclusion Criteria

  • Must have a diagnosis of Fanconi Anemia.
  • Must have one of the following blood-related conditions:
    • Severe Aplastic Anemia with very low bone marrow activity or low blood cell counts, requiring transfusions.
    • Myelodysplastic Syndrome.
    • Acute Myelogenous Leukemia, whether untreated, in remission, or recurring.
  • Donors must be either partially matched relatives or matched unrelated donors.
  • Patients and donors can be of any gender or ethnic background.
  • Must have a performance status score above 70% on specific health scales.
  • Must have adequate heart, liver, kidney, and lung function as measured by specific medical tests.
  • Must be willing to participate in the study and sign a consent form.
  • Female patients and donors must not be pregnant or breastfeeding and must agree to a pregnancy test before the transplant and to avoid pregnancy during the study.

Exclusion Criteria

  • Active leukemia in the central nervous system.
  • Female patients who are pregnant or breastfeeding.
  • Active, uncontrolled infections.
  • Positive test for HIV or HTLV.

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