Myelodysplastic Syndrome Clinical Trial

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

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.

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Full 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.

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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 /= 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

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

200

Study ID:

NCT00619879

Recruitment Status:

Unknown status

Sponsor:

Ann & Robert H Lurie Children's Hospital of Chicago

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There is 1 Location for this study

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Children's Memorial Hospital
Chicago Illinois, 60614, United States More Info
Morris Kletzel, MD
Principal Investigator

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Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

200

Study ID:

NCT00619879

Recruitment Status:

Unknown status

Sponsor:


Ann & Robert H Lurie Children's Hospital of Chicago

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