Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
1 other identifier
observational
81
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Mar 2007
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 26, 2007
CompletedFirst Submitted
Initial submission to the registry
January 8, 2008
CompletedFirst Posted
Study publicly available on registry
February 21, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 7, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
February 7, 2019
CompletedMarch 7, 2025
February 1, 2025
11.9 years
January 8, 2008
March 4, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital.
To study end
Secondary Outcomes (3)
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
Interventions
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. * 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.
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.
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
Eligibility Criteria
All patients that meet the inclusion criteria.
You may qualify if:
- 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).
- rd 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
- +10 more criteria
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Sonali Chaudhury, MD
Ann & Robert H Lurie Children's Hospital of Chicago
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
January 8, 2008
First Posted
February 21, 2008
Study Start
March 26, 2007
Primary Completion
February 7, 2019
Study Completion
February 7, 2019
Last Updated
March 7, 2025
Record last verified: 2025-02