Acute Lymphoblastic Leukemia/LymphomaNCT02338479
Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
An observational study of Acute Lymphoblastic Leukemia/Lymphoma and Myelodysplasia, run by Center for International Blood and Marrow Transplant Research at 32 sites in United States.
Trial summary
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National… Read more about this study
- Who is it for?
- Ages Up to 22 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia/Lymphoma, Myelodysplasia, Acute Myelogenous Leukemia
- How many people
- 340 participants
- Where
- 32 sitesUnited States
- Study dates
- Mar 2015to Dec 2023
- 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 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: This study will enroll is a nonrandomized prospective cohort undergoing HCT for treatment of childhood leukemia and myelodysplasia.
Who may be able to join
- Age less than 22 years at admission for HCT
- Planned allogeneic HCT from any donor and stem cell source. There are no study-specific criteria for HLA-matching
- Disease and disease status criteria
- Acute lymphoblastic leukemia/lymphoma in complete morphologic remission defined as a M1 marrow (<5% blasts) with no evidence of active extramedullary disease within 30 days of the start of the conditioning regimen; OR
- Myelodysplasia (regardless of subtype) with less than 10% marrow blasts within 30 days of the start of the conditioning regimen; OR
- Acute myelogenous leukemia in complete morphologic remission defined as an M1 marrow (<5% blasts) with no evidence of extramedullary disease within 30 days of the start of the conditioning regimen; OR
Show all 16Show fewer
- Juvenile myelomonocytic leukemia; OR
- Chronic myelogenous leukemia excluding refractory blast crisis.
- Planned myeloablative conditioning regimen, defined as a regimen including one of the following as a backbone agent:
- Busulfan ≥ 12.8 mg/kg total dose (IV or PO). PK-based dosing allowed, if the intent is total overall dose ≥ 12.8 mg/kg; OR
- Total Body Irradiation ≥ 1200 cGy fractionated; OR
- Treosulfan ≥ 30 g/m2 total dose IV
- Enrollment in the following NMDP research protocols:
- Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxicity Injuries
- Protocol for a Research Sample Repository for Allogeneic Hematopoietic Stem Cell Transplantation and Marrow Toxic Injuries
- Written informed consent document signed by patient if the age is greater than or equal to 18 years and the patient is developmentally able to provide consent. The informed consent document is to be signed by the parent or legal guardian if the patient's age is less than 18 years or if the patient is older than 18 years, but developmentally unable to provide consent. Assent will be obtained according to the guidelines of the patient's transplant institution.
Who cannot join
- Prior allogeneic or autologous HCT
- Patients with renal disease prior to the start of HCT conditioning requiring the use of dialysis at the time of enrollment and/or GFR < 60 mL/min/1.73 m2
- Patients with osteopenia or osteoporosis treated with a bisphosphonate medication at any time prior to enrollment
- Patients with preexisting diabetes or hyperglycemia treated with insulin or oral hypoglycemic medication at the time of enrollment
- Patients with uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment
- Karnofsky performance score or Lansky Play-Performance Scale Score <60 at the time of study enrollment
- Known inherited or constitutional predisposition to cancer including, but not limited to Down Syndrome, Li-Fraumeni syndrome, Fanconi Anemia, and patients with BRCA1 and BRCA2 mutations
Trial locations and contacts
32 sites in 1 country.
32 sites in 1 country
United States32 sites
University of Michigan
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
University of Colorado
Aurora, Colorado, 80045
Dana Farber Cancer Institute - Pediatrics
Boston, Massachusetts, 02215
Roswell Park Cancer Institute
Buffalo, New York, 14263
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Levine Children's Hospital
Charlotte, North Carolina, 28203
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60601
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
Cleveland Clinic
Cleveland, Ohio, 44195
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center Dallas
Dallas, Texas, 75235
Duke University Medical Center - Pediatrics
Durham, North Carolina, 27705
Indiana University Hospital/Riley Hospital for Children
Indianapolis, Indiana, 46202
The Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Children's Hospital of Los Angeles
Los Angeles, California, 90027
UCLA Center for Health Sciences
Los Angeles, California, 90095
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37235
Children's Hospital & Research Center - Oakland
Oakland, California, 94609
Oregon Health and Science University - Doernbecher Children's Hospital
Portland, Oregon, 97239
Primary Children's Hospital
Salt Lake City, Utah, 84111
Texas Transplant Institute
San Antonio, Texas, 78229
University of California San Francisco Medical Center
San Francisco, California, 94143
Mayo Clinic
Scottsdale, Arizona, 85259-5499
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110
All Children's Hospital
St. Petersburg, Florida, 33701
University of Arizona Medical Center
Tucson, Arizona, 85724
Westchester Medical Center
Valhalla, New York, 10595
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
Read the detailed description
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
The study examines the hypothesis that survivors of pediatric HCT are at risk for late organ toxicity and they will have identifiable biomarkers present within the first two years following HCT which will be predictive for late adverse outcomes allowing for early identification of patients at risk.
- Conditions
- Acute Lymphoblastic Leukemia/Lymphoma, Myelodysplasia, Acute Myelogenous Leukemia, Juvenile Myelomonocytic Leukemia, Chronic Myelogenous Leukemia
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Anemia, Refractory, with Excess of Blasts, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Juvenile, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What is being measured
The primary outcome is the main question the study is designed to answer.
To report the incidence of chronic kidney disease (CKD), metabolic syndrome, and osteopenia
Measured: Baseline to 1 and 2 years following allogeneic HCT for hematologic malignancy
Secondary outcomes (9)
To identify prognostic risk factors for the development and progression of post-HCT CKD, metabolic syndrome, and osteopenia
Measured: Baseline to 1 and 2 years following HCT
To investigate potential associations of systemic hypertension as measured with intermittent blood pressure assessment with proteinuria, acute kidney injury, and CKD
Measured: Baseline to 100 days, and at 1 and 2 years following HCT
To compare the results of GFR estimating equations based on serum cystatin C levels or serum creatinine to GFR measured by nuclear medicine GFR and/or 24-hour creatinine clearance
Measured: Baseline to 180 days, and at 1 and 2 years following HCT
To explore potential association of the protein biomarker elafin in the urine at with the development of CKD
Measured: Baseline to 180 days, and at 1 and 2 years following HCT
To report levels of fasting triglycerides, low-density lipoprotein, high-density lipoprotein, insulin, and glucose levels
Measured: Baseline to 100 days, and at 1 and 2 years following HCT
To assess change in body composition including bone mineral density, body mass index, percent fat mass and lean body mass as measured by dual-energy absorptiometry
Measured: Baseline to 1 and 2 years following HCT
To assess the presence of osteopenia prior to HCT and at 1-year and 2-years following HCT by x-ray in patients unable to undergo DXA without sedation
Measured: Baseline to 1 and 2 years following HCT
To report levels of markers of bone turnover including serum osteocalcin, bone specific alkaline phosphatase, and urine N-telopeptide
Measured: Baseline to 30 days, 100 days, and at 1 and 2 years following HCT
To develop a repository for plasma to be used in future investigation of HCT-associated late effects
Measured: Baseline, 30 days, 100 days, and at 1 and 2 years following HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2015 (estimated)
- Study startMar 2015
- Primary completionSep 2020
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic Leukemia/LymphomaStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 340 participants (actual)
Sponsor and investigators
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Sponsor type
- Research network
- Collaborators
- Pediatric Blood and Marrow Transplant Consortium, National Marrow Donor Program
- Responsible party
- Sponsor
- Investigators
- Christine Duncan, MD, Study chair, Dana-Farber Cancer InstituteK. Scott Baker, MD, Study chair, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02338479
- Sponsor's protocol ID
- 13-TLEC
- Status last verified
- Feb 2023
- First submitted
- 12 Jan 2015
- Last updated
- 8 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT02338479
- Acronym / sponsor ID
- 13-TLEC
- Conditions
- Acute Lymphoblastic Leukemia/Lymphoma, Myelodysplasia, Acute Myelogenous Leukemia, Juvenile Myelomonocytic Leukemia, Chronic Myelogenous Leukemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 340 participants (actual)
- Lead sponsor
- Center for International Blood and Marrow Transplant ResearchResearch network
- Collaborators
- Pediatric Blood and Marrow Transplant Consortium, National Marrow Donor Program
- Study start
- Mar 2015 (actual)
- Primary completion
- Sep 2020 (actual)
- Study completion
- Dec 2023 (estimated)
- First posted
- 14 Jan 2015 (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.
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
Detailed description
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
The study examines the hypothesis that survivors of pediatric HCT are at risk for late organ toxicity and they will have identifiable biomarkers present within the first two years following HCT which will be predictive for late adverse outcomes allowing for early identification of patients at risk.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- This study will enroll is a nonrandomized prospective cohort undergoing HCT for treatment of childhood leukemia and myelodysplasia.
Key inclusion criteria (16 listed)
- Age less than 22 years at admission for HCT
- Planned allogeneic HCT from any donor and stem cell source. There are no study-specific criteria for HLA-matching
- Disease and disease status criteria
- Planned myeloablative conditioning regimen, defined as a regimen including one of the following as a backbone agent:
- Enrollment in the following NMDP research protocols:
Key exclusion criteria (7 listed)
- Prior allogeneic or autologous HCT
- Patients with renal disease prior to the start of HCT conditioning requiring the use of dialysis at the time of enrollment and/or GFR < 60 mL/min/1.73 m2
- Patients with osteopenia or osteoporosis treated with a bisphosphonate medication at any time prior to enrollment
- Patients with preexisting diabetes or hyperglycemia treated with insulin or oral hypoglycemic medication at the time of enrollment
- Patients with uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To report the incidence of chronic kidney disease (CKD), metabolic syndrome, and osteopenia
Time frame: Baseline to 1 and 2 years following allogeneic HCT for hematologic malignancy
9 secondary outcomes
To identify prognostic risk factors for the development and progression of post-HCT CKD, metabolic syndrome, and osteopenia
Time frame: Baseline to 1 and 2 years following HCT
To investigate potential associations of systemic hypertension as measured with intermittent blood pressure assessment with proteinuria, acute kidney injury, and CKD
Time frame: Baseline to 100 days, and at 1 and 2 years following HCT
To compare the results of GFR estimating equations based on serum cystatin C levels or serum creatinine to GFR measured by nuclear medicine GFR and/or 24-hour creatinine clearance
Time frame: Baseline to 180 days, and at 1 and 2 years following HCT
To explore potential association of the protein biomarker elafin in the urine at with the development of CKD
Time frame: Baseline to 180 days, and at 1 and 2 years following HCT
To report levels of fasting triglycerides, low-density lipoprotein, high-density lipoprotein, insulin, and glucose levels
Time frame: Baseline to 100 days, and at 1 and 2 years following HCT
To assess change in body composition including bone mineral density, body mass index, percent fat mass and lean body mass as measured by dual-energy absorptiometry
Time frame: Baseline to 1 and 2 years following HCT
To assess the presence of osteopenia prior to HCT and at 1-year and 2-years following HCT by x-ray in patients unable to undergo DXA without sedation
Time frame: Baseline to 1 and 2 years following HCT
To report levels of markers of bone turnover including serum osteocalcin, bone specific alkaline phosphatase, and urine N-telopeptide
Time frame: Baseline to 30 days, 100 days, and at 1 and 2 years following HCT
To develop a repository for plasma to be used in future investigation of HCT-associated late effects
Time frame: Baseline, 30 days, 100 days, and at 1 and 2 years following HCT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2023.
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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.
Christine Duncan, MD study chair
Dana-Farber Cancer Institute
K. Scott Baker, MD study chair
Fred Hutchinson Cancer Center
32 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Lymphoblastic Leukemia/Lymphoma and Myelodysplasia, run by Center for International Blood and Marrow Transplant Research at 32 sites 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 16 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites 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?
Results are not available in the source record.
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?
To report the incidence of chronic kidney disease (CKD), metabolic syndrome, and osteopenia (time frame: Baseline to 1 and 2 years following allogeneic HCT for hematologic malignancy). 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 8 Feb 2023.