Back to results

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Primary Purpose

Leukemia, Myelogenous, Chronic, Leukemia, Lymphoblastic, Acute, Leukemia, Myelogenous, Acute

Status

Unknown status

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients

Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies

Hematopoietic Progenitor Cell Transplantation (HPCT)

CNS radiation treatment for ALL with prior CNS disease patients

About this trial

This is an interventional treatment trial for Leukemia, Myelogenous, Chronic focused on measuring 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

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept 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.

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

Sites / Locations

Children's Memorial HospitalRecruiting

Outcomes

Primary Outcome Measures

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

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.

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.

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.

Full Information

NCT ID

NCT00619879

First Posted

January 8, 2008

Last Updated

March 29, 2011

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

1. Study Identification

Unique Protocol Identification Number

NCT00619879

Brief Title

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Official Title

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

Study Type

Interventional

2. Study Status

Record Verification Date

March 2011

Overall Recruitment Status

Unknown status

Study Start Date

March 2007 (undefined)

Primary Completion Date

January 2020 (Anticipated)

Study Completion Date

January 2020 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

4. Oversight

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

200 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies

Intervention Description

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. Pharmacokinetic analysis will guide dose modifications targeted to receive an average AUC of 800-1200 microMols*min for the 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. Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3 and -2.

Intervention Type

Other

Intervention Name(s)

Hematopoietic Progenitor Cell Transplantation (HPCT)

Intervention Description

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.

Intervention Type

Radiation

Intervention Name(s)

CNS radiation treatment for ALL with prior CNS disease patients

Intervention Description

Patients with prior CNS disease over the age of 1 year will be treated with 600 cGy of cranial irradiation in addition to 1200 cGy of TBI. Patients diagnosed with ALL with CNS disease (at the time of diagnosis or relapse) < 1 year of age will receive CNS treatment as Intrathecal Methotrexate as follows: Infants ≤ 1 year of age at the time of Intrathecal Therapy will receive a dosing of 7.5 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count Children 1-2 years of age at the time of Intrathecal Therapy will receive 8 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count

Primary Outcome Measure Information:

Title

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

Time Frame

To study end

Secondary Outcome Measure Information:

Title

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

Title

Time Frame

To study end

Title

Time Frame

To study end

10. Eligibility

Sex

All

Maximum Age & Unit of Time

21 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Morris Kletzel, MD

Phone

773-880-4562

mkletzel@northwestern.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Terriss Conterato

Phone

773-880-8153

TConterato@childrensmemorial.org

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Morris Kletzel, MD

Organizational Affiliation

Ann & Robert H Lurie Children's Hospital of Chicago

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Memorial Hospital

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60614

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Morris Kletzel, MD

12. IPD Sharing Statement

Learn more about this trial

Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies

We'll reach out to this number within 24 hrs