Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies - Full Text View

Sponsor:	Children's Memorial Hospital
Information provided by:	Children's Memorial Hospital
ClinicalTrials.gov Identifier:	NCT00619879

Purpose

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	Intervention	Phase
Leukemia, Myelogenous, Chronic Leukemia, Lymphoblastic, Acute Leukemia, Myelogenous, Acute Juvenile Myelomonocytic Leukemia Dysmyelopoietic Syndromes	Drug: Myeloablative Chemotherapy Regimen for Lymphoid Malignancies Drug: Myeloablative Chemotherapy Regimen for Myeloid Malignancies Other: Hematopoietic Progenitor Cell Transplanation (HPCT)	Phase III

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Resource links provided by NLM:

MedlinePlus related topics: Cancer Leukemia, Adult Acute Leukemia, Adult Chronic Leukemia, Childhood

Drug Information available for: Cyclophosphamide Busulfan Etoposide

U.S. FDA Resources

Further study details as provided by Children's Memorial Hospital:

Primary Outcome Measures:

Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital. [ Time Frame: To study end ] [ Designated as safety issue: Yes ]

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. [ Time Frame: To study end ] [ Designated as safety issue: Yes ]
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 ] [ Designated as safety issue: Yes ]
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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	200
Study Start Date:	March 2007
Estimated Study Completion Date:	January 2020
Estimated Primary Completion Date:	January 2020 (Final data collection date for primary outcome measure)

Intervention Details:

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.

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.

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.

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.

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 either total body irradiation, etoposide, and cyclophosphamide, or busulfan and cyclophosphamide, for patients with transplant eligible lumphoid malignant conditions. The study will accrue patients until a superior method of HPCT is determined and set forth as the new standard of care.

Eligibility

Ages Eligible for Study:	up to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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)
  - Acute nonlymphoblastic leukemia (ANLL) in 2nd remission/early relapse or with other high risk features and the approval of the PI.
- Myelodysplastic/Myeloproliferative Disease
  - Juvenile Myelomonocytic Leukemia (JMML)
  - Myelosplastic syndrome and/or pre-leukemia at any stage
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; Karnofsy status for those > 16 years of age >/= 70

Exclusion Criteria:

Patients who are pregnant
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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00619879

Contacts

Contact: Morris Kletzel, MD	773-880-4562	mkletzel@northwestern.edu
Contact: Terriss Conterato	773-880-8153	TConterato@childrensmemorial.org

Locations

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

Sponsors and Collaborators

Children's Memorial Hospital

Investigators

Principal Investigator:

Morris Kletzel, MD

Children's Memorial Hospital

More Information

No publications provided

Responsible Party:	Children's Memorial Hospital ( Morris Kletzel, MD )
Study ID Numbers:	SCT 0307
Study First Received:	January 8, 2008
Last Updated:	August 26, 2008
ClinicalTrials.gov Identifier:	NCT00619879 History of Changes
Health Authority:	United States: Institutional Review Board

Keywords provided by Children's Memorial Hospital:

Leukemia, Myelogenous, Chronic
Leukemia, Lymphoblastic, Acute
Leukemia, Myelogenous, Acute
Myeloproliferative-Myelodisplastic Diseases
Juvenile Myelomonocytic Leukemia
Dysmyelopoietic Syndromes
Stem Cell Transplantation, Hematopoietic
Allogeneic Transplantation

Human Leukocyte Antigens
Busulfan
Total Body Irradiation
VP-16
Etoposide
Cyclophosphamide
Graft-Versus-Host Disease

Additional relevant MeSH terms:

Leukemia, Lymphoid
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Precancerous Conditions
Antineoplastic Agents
Physiological Effects of Drugs
Cyclophosphamide
Leukemia, Myeloid, Acute
Leukemia
Preleukemia
Pathologic Processes
Therapeutic Uses
Syndrome
Alkylating Agents
Neoplasms by Histologic Type

Precursor Cell Lymphoblastic Leukemia-Lymphoma
Immunoproliferative Disorders
Disease
Immune System Diseases
Hematologic Diseases
Leukemia, Myelomonocytic, Chronic
Myelodysplastic Syndromes
Myeloproliferative Disorders
Leukemia, Myelomonocytic, Juvenile
Leukemia, Myeloid
Immunosuppressive Agents
Pharmacologic Actions
Leukemia, Myelomonocytic, Acute
Lymphatic Diseases
Neoplasms

ClinicalTrials.gov processed this record on February 08, 2010