Clinical trial NCT00619879: Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
Official title: Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Leukemia, Myelogenous, Chronic
Leukemia, Lymphoblastic, Acute
Leukemia, Myelogenous, Acute
Myeloproliferative-Myelodysplastic Diseases
Lymphoma, Malignant
Keywords: Leukemia, Myelogenous, Chronic
Leukemia, Lymphoblastic, Acute
Leukemia, Myelogenous, Acute
Myeloproliferative-Myelodysplastic Diseases
Juvenile Myelomonocytic Leukemia
Dysmyelopoietic Syndromes
Lymphoma, Malignant
Stem Cell Transplantation, Hematopoietic
Allogeneic Transplantation
Human Leukocyte Antigens
Busulfan
Total Body Irradiation
VP-16
Etoposide
Cyclophosphamide
Graft-Versus-Host Disease
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2008-02-21
Enrollment: 81
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients that meet the inclusion criteria.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital.
timeFrame: To study end
Secondary outcomes:
measure: 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.
timeFrame: To study end
measure: 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.
timeFrame: To study end
measure: 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.
timeFrame: To study end
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Sonali Chaudhury, MD
role: PRINCIPAL_INVESTIGATOR
References: