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Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies

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ClinicalTrials.gov Identifier: NCT02338479
Recruitment Status : Active, not recruiting
First Posted : January 14, 2015
Last Update Posted : May 16, 2019
Sponsor:
Collaborators:
Pediatric Blood and Marrow Transplant Consortium
National Marrow Donor Program
Information provided by (Responsible Party):
Center for International Blood and Marrow Transplant Research

Tracking Information
First Submitted Date January 12, 2015
First Posted Date January 14, 2015
Last Update Posted Date May 16, 2019
Actual Study Start Date March 2015
Estimated Primary Completion Date February 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 12, 2015)
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 ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: January 12, 2015)
  • 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 ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
Official Title Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
Brief 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 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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population This study will enroll is a nonrandomized prospective cohort undergoing HCT for treatment of childhood leukemia and myelodysplasia.
Condition
  • Acute Lymphoblastic Leukemia/Lymphoma
  • Myelodysplasia
  • Acute Myelogenous Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Chronic Myelogenous Leukemia
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: January 12, 2015)
340
Original Estimated Enrollment Same as current
Estimated Study Completion Date February 2020
Estimated Primary Completion Date February 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  1. Age less than 22 years at admission for HCT
  2. Planned allogeneic HCT from any donor and stem cell source. There are no study-specific criteria for HLA-matching
  3. Disease and disease status criteria

    1. 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
    2. Myelodysplasia (regardless of subtype) with less than 10% marrow blasts within 30 days of the start of the conditioning regimen; OR
    3. 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
    4. Juvenile myelomonocytic leukemia; OR
    5. Chronic myelogenous leukemia excluding refractory blast crisis.
  4. Planned myeloablative conditioning regimen, defined as a regimen including one of the following as a backbone agent:

    1. 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
    2. Total Body Irradiation ≥ 1200 cGy fractionated; OR
    3. Treosulfan ≥ 30 g/m2 total dose IV
  5. Enrollment in the following NMDP research protocols:

    1. Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxicity Injuries
    2. Protocol for a Research Sample Repository for Allogeneic Hematopoietic Stem Cell Transplantation and Marrow Toxic Injuries
  6. 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.

Exclusion Criteria:

  1. Prior allogeneic or autologous HCT
  2. 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
  3. Patients with osteopenia or osteoporosis treated with a bisphosphonate medication at any time prior to enrollment
  4. Patients with preexisting diabetes or hyperglycemia treated with insulin or oral hypoglycemic medication at the time of enrollment
  5. Patients with uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment
  6. Karnofsky performance score or Lansky Play-Performance Scale Score <60 at the time of study enrollment
  7. 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 22 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02338479
Other Study ID Numbers 13-TLEC
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Center for International Blood and Marrow Transplant Research
Study Sponsor Center for International Blood and Marrow Transplant Research
Collaborators
  • Pediatric Blood and Marrow Transplant Consortium
  • National Marrow Donor Program
Investigators
Study Chair: Christine Duncan, MD Dana-Farber Cancer Institute
Study Chair: K. Scott Baker, MD Fred Hutchinson Cancer Research Center
PRS Account Center for International Blood and Marrow Transplant Research
Verification Date May 2019