Clinical Trials /

Immunoablative Mini Transplant (Hematopoietic Peripheral Blood Stem Cell Transplant [HPBSC])

NCT00179764

Description:

The purpose of this research study is to evaluate the effectiveness of transplantation of high doses of peripheral blood stem cells (stem cells are special cells found in the blood and bone marrow that produce new blood cells) after treatment with non-myeloablative chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side effects of the transplant.

Related Conditions:
  • Acute Lymphoblastic Leukemia
Recruiting Status:

Unknown status

Phase:

Phase 2

Trial Eligibility

Document

Immunoablative Mini Transplant (Hematopoietic Peripheral Blood <span class="go-doc-concept go-doc-intervention">Stem Cell Transplant</span> [HPBSC])

Title

  • Brief Title: Immunoablative Mini Transplant (Hematopoietic Peripheral Blood Stem Cell Transplant [HPBSC])
  • Official Title: Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
  • Clinical Trial IDs

    NCT ID: NCT00179764

    ORG ID: BMT 0300 Mini

    Trial Conditions

    Tumors

    Malignant Melanoma

    Hematological Malignancies

    Myelogenous Leukemia, Chronic

    Leukemia, Lymphoblastic, Acute

    Trial Interventions

    Drug Synonyms Arms

    Trial Purpose

    The purpose of this research study is to evaluate the effectiveness of transplantation of
    high doses of peripheral blood stem cells (stem cells are special cells found in the blood
    and bone marrow that produce new blood cells) after treatment with non-myeloablative
    chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side
    effects of the transplant.

    Detailed Description

    The standard treatment in many disorders of the bone marrow is high dose chemotherapy and
    whole-body radiation treatment followed by the stem cell transplant. This type of
    transplant not only suppresses or kills off the immune system, but is very toxic to the bone
    marrow. This study uses a chemotherapy regimen that will suppress the patient's immune
    system; however, it is non-myeloablative (not toxic to the bone marrow). It does not use
    whole-body radiation treatment. This approach can minimize the short- and long-term effects
    of transplantation. Other studies have shown that using chemotherapy followed by bone
    marrow transplantation without whole-body radiation can produce similar results as treatment
    with whole-body radiation.

    Patients will be given chemotherapy with Fludarabine and Busulfan prior to the stem cell
    transplant. This treatment not only destroys diseased cells, but it also kills normal bone
    marrow cells. Following this experimental treatment, the patient will be given the stem
    cells through a central venous catheter (tube inserted in a vein). When the healthy stem
    cells are given to the patient, they will replace the destroyed bone marrow cells and
    produce new blood cells. The Allogeneic (not one's own) stem cells used in this
    experimental transplant will be obtained from a related matched donor or from an unrelated
    matched donor located through the National Marrow Donor Program.

    Trial Arms

    Name Type Description Interventions

    Eligibility Criteria

    Inclusion Criteria:

    - Patients with recurrent solid tumors

    - Patients with malignant melanoma

    - Patients with hematological malignancies.

    - Chronic myelogenous leukemia in chronic or accelerated phase, to include chronic
    myelomonocytic leukemia (juvenile chronic myelogenous leukemia [JCML] or CMML).

    - Acute lymphoblastic leukemia (ALL)

    - First remission high-risk ALL (Ph+ with initial high white blood cell
    [WBC]; t (4-11) in infants less than 1 year and CALLA negative)

    - Second or subsequent remission ALL or isolated extramedullary disease on or
    off therapy.

    - Acute non-lymphocytic leukemia (ANLL)

    - Patients with ANLL in first remission who have a matched sibling donor.

    - ANLL in second remission, or patients who only achieve an initial partial
    remission < 15% blasts, or early relapse.

    - Myelodysplastic syndromes (MDS): refractory anemia (RA), refractory anemia with
    excess blasts (RAEB), refractory anemia with excess blasts in transformation
    (RAEB-T) and CMML/JCML.

    - Selected immunodeficiencies:

    - Wiskott-Aldrich syndrome.

    - Severe combined immunodeficiency variants that require ablation.

    - Hyper-IGM syndrome.

    - Other immune deficiencies after approval from the medical director.

    - Bone marrow failure syndromes (single or multiple hematopoietic lines)

    - Venous access: A double lumen central vascular access device or its equivalent will
    be required for all patients entered on the protocol.

    - Informed consent: The donor and the patient and/or the patient's legally authorized
    guardian must acknowledge in writing that consent to become a study subject has been
    obtained in accordance with the institutional policy approved by the United States
    (U.S.) Department of Health and Human Services.

    - Patient organ function requirements:

    - Adequate renal function: serum creatinine < 2 x normal, or creatinine clearance
    calculated by Schwartz formula, of glomerular filtration rate (GFR) > 40
    ml/min/1.73m2, or an equivalent GFR as determined by the institutional normal
    range.

    - Adequate liver function: total bilirubin </= 2 x normal; and SGOT (AST) or SGPT
    (ALT) </= 4 x normal.

    - Adequate cardiac function: shortening fraction of > 24% by echocardiogram, or
    ejection fraction of > 30% by radionuclide angiogram.

    - Adequate pulmonary function: DLCO, FEV1 / FVC > 30% by pulmonary function test.
    For children who are uncooperative for pulmonary function tests and have no
    evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on
    room air is considered acceptable.

    - Performance status: Lansky >/= 60% for children </= 16 years of age; or
    Karnofsky > 60% status for those > 16 years of age.

    Exclusion Criteria:

    - Patients who are pregnant

    - Inability to find a suitable donor for the patient

    - Patient is HIV-positive

    - Patient has active Hepatitis B

    - Disease progression or relapse prior to HPC infusion

    Minimum Eligible Age: N/A

    Maximum Eligible Age: 21 Years

    Eligible Gender: Both

    Primary Outcome Measures

    Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ rich HPCs after a reduced intensity conditioning regimen.

    Determine the toxicity of a reduced intensity conditioning regimen consisting of Fludarabine and Busulfan.

    Secondary Outcome Measures

    Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples.

    Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders.

    Assess the relapse rate of patients transplanted with this reduced intensity regimen.

    Determine the incidence of acute and chronic Graft vs. Host Disease (GVHD) using prophylaxis with Cyclosporine A and mycophenolate mofetil following this reduced intensity regimen.

    Trial Keywords

    Patients with recurrent solid tumors

    Patients with malignant melanoma

    Patients with hematological malignancies

    Acute lymphoblastic leukemia (ALL)

    Acute myelogenous leukemia (AML)