Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago (Other)

Overall Status

Unknown status

CT.gov ID

NCT00619879

Collaborator

(none)

200

Enrollment

Location

154.1

Duration (Months)

1.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Leukemia, Myelogenous, Chronic Leukemia, Lymphoblastic, Acute Leukemia, Myelogenous, Acute Myeloproliferative-Myelodysplastic Diseases Lymphoma, Malignant	Drug: Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients Drug: Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies Other: Hematopoietic Progenitor Cell Transplantation (HPCT) Radiation: CNS radiation treatment for ALL with prior CNS disease patients	Phase 3

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 Design

Study Type:

Interventional

Anticipated Enrollment :

200 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Study Start Date :

Mar 1, 2007

Anticipated Primary Completion Date :

Jan 1, 2020

Anticipated Study Completion Date :

Jan 1, 2020

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 21 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.