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

Status unconfirmed

Conditions studied: Acute Lymphoblastic Leukemia/Lymphoma, Myelodysplasia, Acute Myelogenous Leukemia, Juvenile Myelomonocytic Leukemia, Chronic Myelogenous Leukemia

In brief

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.

Key facts

Study ID: NCT02338479
Run by: Center for International Blood and Marrow Transplant Research
People needed: 340
Starts: 2015-03-01
Expected to finish: 2023-12-01
Last updated by the study team: 2023-02-08

Who can join

Age: any, up to 22. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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
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.

You may not qualify if…

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

Where it is running

Mayo Clinic — Scottsdale, Arizona, United States
University of Arizona Medical Center — Tucson, Arizona, United States
Children's Hospital of Los Angeles — Los Angeles, California, United States
UCLA Center for Health Sciences — Los Angeles, California, United States
Children's Hospital & Research Center - Oakland — Oakland, California, United States
University of California San Francisco Medical Center — San Francisco, California, United States
University of Colorado — Aurora, Colorado, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
All Children's Hospital — St. Petersburg, Florida, United States
Children's Healthcare of Atlanta — Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
Indiana University Hospital/Riley Hospital for Children — Indianapolis, Indiana, United States
Dana Farber Cancer Institute - Pediatrics — Boston, Massachusetts, United States
University of Michigan — Ann Arbor, Michigan, United States
The Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States
Washington University/St. Louis Children's Hospital — St Louis, Missouri, United States
Roswell Park Cancer Institute — Buffalo, New York, United States
Westchester Medical Center — Valhalla, New York, United States
University of North Carolina Hospitals — Chapel Hill, North Carolina, United States
Levine Children's Hospital — Charlotte, North Carolina, United States
Duke University Medical Center - Pediatrics — Durham, North Carolina, United States
University Hospitals Case Medical Center — Cleveland, Ohio, United States
Cleveland Clinic — Cleveland, Ohio, United States
Nationwide Children's Hospital — Columbus, Ohio, United States
Oregon Health and Science University - Doernbecher Children's Hospital — Portland, Oregon, United States

Full record on ClinicalTrials.gov

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