Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Phase 3

Completed

• Conditions: Leukemia, Myelogenous, Chronic; Leukemia, Lymphoblastic, Acute; Leukemia, Myelogenous, Acute; Myeloproliferative-Myelodysplastic Diseases; Lymphoma, Malignant

• Registration Number: NCT00619879
• Lead Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

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 Timeline

• Study Start: 2007-03-26
• Study First Submitted: 2008-01-08
• Study First Submitted QC: 2008-02-07
• Study First Posted: 2008-02-21
• Primary Completion: 2019-02-07
• Study Completion: 2019-02-07
• Status Verified: 2025-02
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 81

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital.	To study end

Secondary Outcome Measures

Name	Time	Method
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.	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.	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.	To study end

Trial Locations

Locations (1)

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States