Leukemia, Myelogenous, ChronicNCT00619879
Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
An observational study of Leukemia, Myelogenous, Chronic and Leukemia, Lymphoblastic, Acute, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Leukemia, Myelogenous, Chronic, Leukemia, Lymphoblastic, Acute, Leukemia, Myelogenous, Acute
- What is being tested?
- Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients, Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies and Hematopoietic Progenitor Cell Transplantation (HPCT)
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 81 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Mar 2007to 7 Feb 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All patients that meet the inclusion criteria.
Who may be able to join
- 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.
Show all 25Show fewer
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
About this study
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.
Read the 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.
- Conditions
- Leukemia, Myelogenous, Chronic, Leukemia, Lymphoblastic, Acute, Leukemia, Myelogenous, Acute, Myeloproliferative-Myelodysplastic Diseases, Lymphoma, Malignant
- Being tested
- 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
- 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
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic-Myeloproliferative Diseases, Lymphoma, Leukemia, Myelomonocytic, Juvenile, Myelodysplastic Syndromes, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit RecipientsDrug
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.
Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid MalignanciesDrug
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 Cell Transplantation (HPCT)Other
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.
CNS radiation treatment for ALL with prior CNS disease patientsRadiation
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
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: To study end
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2008 (estimated)
- Study start26 Mar 2007
- Primary completion7 Feb 2019
- Study completion7 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myelogenous, ChronicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sonali Chaudhury, MD, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Sonali Chaudhury, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00619879
- Sponsor's protocol ID
- SCT 0307
- Status last verified
- Feb 2025
- First submitted
- 8 Jan 2008
- Last updated
- 7 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies
- ClinicalTrials.gov ID
- NCT00619879
- Acronym / sponsor ID
- SCT 0307
- Conditions
- 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
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- 26 Mar 2007 (actual)
- Primary completion
- 7 Feb 2019 (actual)
- Study completion
- 7 Feb 2019 (actual)
- First posted
- 21 Feb 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All patients that meet the inclusion criteria.
Key inclusion criteria (25 listed)
- Malignant Disease
- 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:
- Performance status: Lansky for children </= 16 years >/= 60; Karnofsky status for those > 16 years of age >/= 70
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit RecipientsTotal 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. | Drug | – |
| Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid MalignanciesBusulfan 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. | Drug | – |
| Hematopoietic Progenitor Cell Transplantation (HPCT)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. | Other | – |
| CNS radiation treatment for ALL with prior CNS disease patientsPatients 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 | Radiation | – |
Primary outcome
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital.
Time frame: To study end
3 secondary outcomes
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
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.
Time frame: 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.
Time frame: To study end
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sonali Chaudhury, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Myelogenous, Chronic and Leukemia, Lymphoblastic, Acute, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital. (time frame: To study end). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2025.