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Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

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ClinicalTrials.gov Identifier: NCT00619879
Recruitment Status : Unknown
Verified March 2011 by Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was:  Recruiting
First Posted : February 21, 2008
Last Update Posted : March 30, 2011
Sponsor:
Information provided by:
Ann & Robert H Lurie Children's Hospital of Chicago

Tracking Information
First Submitted Date  ICMJE January 8, 2008
First Posted Date  ICMJE February 21, 2008
Last Update Posted Date March 30, 2011
Study Start Date  ICMJE March 2007
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 7, 2008)
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital. [ Time Frame: To study end ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 29, 2011)
  • Evaluate the effectiveness of graft versus host disease prevention with a combination of anti-thymocyte globulin, continuous infusion cyclosporine, and short course methotrexate for transplants. [ Time Frame: To study end ]
  • Determine the toxicity of a single conditioning regimen consisting of total body irradiation, etoposide, and Cyclophosphamide for patients with transplant eligible lymphoid malignant conditions or myeloid malignant conditions receiving cord blood units. [ Time Frame: To study end ]
  • Determine the toxicity of a single conditioning regimen consisting of Busulfan and Cyclophosphamide for patients with transplant eligible myeloid malignant conditions who are not receiving cord blood units. [ Time Frame: To study end ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 7, 2008)
  • Evaluate the effectiveness of graft versus host disease prevention with a combination of anti-thymocyte globulin, continuous infusion cyclosporine, and short course methotrexate for transplants. [ Time Frame: To study end ]
  • Determine the toxicity of a single conditioning regimen consisting of total body irradiation, etoposide, and cyclophosphamide for patients with transplant eligible lymphoid malignant conditions. [ Time Frame: To study end ]
  • Determine the toxicity of a single conditioning regimen consisting of busulfan and cyclophosphamide for patients with transplant eligible myeloid malignant conditions. [ Time Frame: To study end ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
Official Title  ICMJE Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
Brief Summary The purpose of this study is to show that myeloablative hematopoietic progenitor cell transplantation (HPCT) continues to offer acceptable disease-free survival for select patients requiring HPCT.
Detailed Description Myeloablative hematopoietic progenitor cell transplantation (HPCT) remains the standard of care for patients requiring HPCT. The purpose of this study is to evaluate the morbidity and mortality of myeloablative HPCT at Children's Memorial Hospital. It will also look to determine the toxicity of a single conditioning regimen consisting of total body irradiation (TBI), etoposide (VP-16), and Cyclophosphamide for patients with transplant eligible lymphoid malignant conditions or with transplant eligible myeloid malignant conditions who are receiving cord blood units, or to determine the toxicity of a single conditioning regimen consisting of Busulfan and Cyclophosphamide for patients with transplant eligible myeloid malignant conditions who are not receiving cord blood units.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Leukemia, Myelogenous, Chronic
  • Leukemia, Lymphoblastic, Acute
  • Leukemia, Myelogenous, Acute
  • Myeloproliferative-Myelodysplastic Diseases
  • Lymphoma, Malignant
Intervention  ICMJE
  • Drug: Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients

    Total Body Irradiation (TBI) 1200 cGy will be given on days -8,-7,-6 and -5 in eight sessions, delivering 150cGy in each session.

    Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4.

    Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3, -2.

  • Drug: Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies

    Busulfan administration:

    • For children >/= 4 years of age, Busulfan 0.8 mg/kg/dose will be given every 6 hours over days -8,-7, -6, and -5 for a total of 16 doses.
    • For children < 4 years of age, Busulfan 1 mg/kg/dose will be given every 6 hours over days -8, -7, -6, -5 for a total of 16 doses.
    • Pharmacokinetic analysis will guide dose modifications targeted to receive an average AUC of 800-1200 microMols*min for the 16 doses.

    Lorazepam (0.05 mg/kg) IV will be administered one half hour before the initial dose of Busulfan is given and every 6 hours through day -4.

    Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4.

    Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3 and -2.

  • Other: Hematopoietic Progenitor Cell Transplantation (HPCT)
    Hematopoietic progenitor cells (HPCs) will be infused on day 0. Source of cells may be bone marrow, peripheral blood cells, or cord blood units, from matched related or unrelated donors.
  • Radiation: CNS radiation treatment for ALL with prior CNS disease patients

    Patients with prior CNS disease over the age of 1 year will be treated with 600 cGy of cranial irradiation in addition to 1200 cGy of TBI.

    Patients diagnosed with ALL with CNS disease (at the time of diagnosis or relapse) < 1 year of age will receive CNS treatment as Intrathecal Methotrexate as follows:

    • Infants ≤ 1 year of age at the time of Intrathecal Therapy will receive a dosing of 7.5 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count
    • Children 1-2 years of age at the time of Intrathecal Therapy will receive 8 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: February 7, 2008)
200
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2020
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Malignant Disease

    • Chronic myleogenous leukemia in chronic or accelerated phase
    • Acute lymphoblastic leukemia (ALL)

      • First remission high-risk ALL (Ph+, t( 4-11) infants).
      • Second remission ALL, after a short first remission (<36 mos from Dx).
      • 3rd or greater remission ALL.
    • Acute myelogenous leukemia (AML)

      • First remission high risk acute nonlymphoblastic (ANLL) (as defined by cytogenetics), if a matched sibling donor is available.
      • Initial partial remission AML (<20% blasts in the bone marrow).
      • AML that is refractory to two cycles of induction therapy.
      • Second or greater remission AML
    • Myelodysplastic/Myeloproliferative Disease

      • Juvenile Myelomonocytic Leukemia (JMML)
      • Myelosplastic syndrome and/or pre-leukemia at any stage
    • Lymphoma

      • Relapsed lymphoma with residual disease that appears to be chemo-sensitive and non-bulky (<5 cm at largest diameter)
  • Venous Access: Three lumens of central vascular access will be required for all patients entered on protocol due to the need for a dedicated line for continuous infusion cyclosporine.
  • Informed Consent: 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 policies approved by the U.S. Department of Health and Human Services.
  • Patient organ function requirements:

    • Adequate renal function: Serum Creatinine <~1.5 x normal, or Creatinine clearance of 70 mL/min/1.73 mE2 or an equivalent GFR as determined by the institutional normal range
    • Adequate liver function: Total bilirubin <1.5 x normal; and SGOT (AST) or SGPT (ALT) <~2.5 x normal
    • Adequate cardiac function: Shortening fraction of >/=27% by echocardiogram
    • Adequate pulmonary function: FEV1/FVC >/=60% by pulmonary function test; for children who are uncooperative, no evidence of dysnpea at rest, or exercise intolerance, and must have a pulse oximetry >94% in room air
  • Performance status: Lansky for children </= 16 years >/= 60; Karnofsky status for those > 16 years of age >/= 70
  • Effective Contraceptive Use: Women of childbearing potential and sexually active males should use effective contraception while on study.

Exclusion Criteria:

  • Patients who are pregnant or lactating
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00619879
Other Study ID Numbers  ICMJE SCT 0307
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Morris Kletzel, MD, Children's Memorial Hospital
Study Sponsor  ICMJE Ann & Robert H Lurie Children's Hospital of Chicago
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Morris Kletzel, MD Ann & Robert H Lurie Children's Hospital of Chicago
PRS Account Ann & Robert H Lurie Children's Hospital of Chicago
Verification Date March 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP