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
  • Acute Myeloid Leukemia
  • Chronic Myelomonocytic Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Melanoma
  • Myelodysplastic Syndromes
  • Refractory Anemia
  • Refractory Anemia with Excess Blasts
  • Solid Neoplasm
Recruiting Status:

Completed

Phase:

N/A

Trial Eligibility

Document

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

  • ORG STUDY ID: BMT 0300 Mini
  • NCT ID: NCT00179764

Conditions

  • Tumors
  • Malignant Melanoma
  • Hematological Malignancies
  • Myelogenous Leukemia, Chronic
  • Leukemia, Lymphoblastic, Acute

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

NameTypeDescriptionInterventions

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-Immunoglobulin M (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 Aspartate
                  aminotransferase (AST) or Alanine aminotransferase (ALT) </= 4 x normal.

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

               -  Adequate pulmonary function: Diffusion Lung Capacity Carbon Monoxide (DLCO),
                  Forced Expiratory Volume in 1 second (FEV1) / Forced Vital Capacity (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 Score >/= 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
      
Maximum Eligible Age:21 Years
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ HPCs after a reduced intensity conditioning regimen.
Time Frame:To study end
Safety Issue:
Description:

Secondary Outcome Measures

Measure:Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples.
Time Frame:To study end
Safety Issue:
Description:
Measure:Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders.
Time Frame:To study end
Safety Issue:
Description:
Measure:Assess the relapse rate of patients transplanted with this reduced intensity regimen.
Time Frame:To study end
Safety Issue:
Description:
Measure: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.
Time Frame:To study end
Safety Issue:
Description:

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Unknown status
Lead Sponsor:Ann & Robert H Lurie Children's Hospital of Chicago

Trial Keywords

  • Patients with recurrent solid tumors
  • Patients with malignant melanoma
  • Patients with hematological malignancies
  • Acute lymphoblastic leukemia (ALL)
  • Acute myelogenous leukemia (AML)

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