List All Bone Marrow Transplant Trials

Bone Marrow Transplant

Allogeneic HCT for Nonmalignant Inherited Disorders w/ Treosulfan (FHCRC-2256)
Allogeneic Hematopoietic Cell Transplantation for Patients with Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen
Status Conditions Phase Study ID
Recruiting Dyskeratosis Congenita
Graft Versus Host Disease
Nonneoplastic Condition
Shwachman-Diamond Syndrome
Phase II FHCRC-2256

RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus, methotrexate, cyclosporine, and mycophenolate mofetil before and after transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well giving treosulfan together with fludarabine phosphate followed by donor stem cell transplant works in treating patients with noncancerous inherited disorders.

Lauri Burroughs, MD
Seattle Cancer Care Alliance 800-804-8824  
Eligibility Criteria (must meet the following to participate in this study)
Ages Eligible for Study:   up to 54 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Diagnosed with nonmalignant inherited disease, including any of the following:

    • Bone marrow failure syndrome

      • Dyskeratosis congenita
      • Shwachman-Diamond syndrome
    • Immunodeficiency disease

      • Chronic granulomatous disease
      • Leukocyte adhesion defect
      • Wiskott-Aldrich syndrome
      • Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX)
      • CD40-ligand deficiency
    • Hemophagocytosis lymphohistiocytosis
    • Metabolic storage disease

      • Mucopolysaccharidosis
      • Sphingolipidoses
    • Osteopetrosis
  • No aplastic anemia or Fanconi anemia
  • Disease considered treatable by allogeneic hematopoietic stem cell transplantation AND has one of the following donors available:

    • Bone marrow or peripheral blood stem cell donor meeting 1 of the following criteria:

      • HLA-identical related donor
      • Unrelated donor matched at HLA-A, B, C, DRB1, and DQB1 OR mismatched for a single allele at HLA-A, B, C, or DRB1 or a single antigen or allele at DQB1 by high-resolution DNA typing
    • Umbilical cord blood (UCB) donor meeting the following criteria:

      • UCB unit(s) must be matched with the recipient at ≥ 4 of 6 HLA-A, B, and DRB1 loci
      • For patients receiving two UCB units, the UCB units must also be matched to each other at ≥ 3 of 6 HLA-A, B, and DRB1 loci

        • Each UCB unit must contain ≥ 1.5 x 10^7 total nucleated cell (TNC) per kilogram recipient weight
        • Total cell dose of the combined units must be ≥ 3.0 x 10^7 TNC per kilogram recipient weight
Last Updated
May 27, 2010
See this trial at
Access protocol and consent forms at Fred Hutchinson Cancer Research Center
Disclaimer: We update this information regularly. However, what you read today may not be completely up to date.

Please remember:
  • Talk to your health care providers first before making decisions about your health care.
  • Whether you are eligible for a research study depends on many things. There are specific requirements to be in research studies. These requirements are different for each study.