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

NCT ID: NCT02338479

Last Updated: 2023-02-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

340 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-03-31

Study Completion Date

2023-12-31

Brief Summary

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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

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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

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Acute Lymphoblastic Leukemia/Lymphoma Myelodysplasia Acute Myelogenous Leukemia Juvenile Myelomonocytic Leukemia Chronic Myelogenous Leukemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Maximum Eligible Age

22 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pediatric Blood and Marrow Transplant Consortium

OTHER

Sponsor Role collaborator

National Marrow Donor Program

OTHER

Sponsor Role collaborator

Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Christine Duncan, MD

Role: STUDY_CHAIR

Dana-Farber Cancer Institute

K. Scott Baker, MD

Role: STUDY_CHAIR

Fred Hutchinson Cancer Center

Locations

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Mayo Clinic

Scottsdale, Arizona, United States

Site Status

University of Arizona Medical Center

Tucson, Arizona, United States

Site Status

Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

UCLA Center for Health Sciences

Los Angeles, California, United States

Site Status

Children's Hospital & Research Center - Oakland

Oakland, California, United States

Site Status

University of California San Francisco Medical Center

San Francisco, California, United States

Site Status

University of Colorado

Aurora, Colorado, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Indiana University Hospital/Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Dana Farber Cancer Institute - Pediatrics

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

The Children's Mercy Hospitals and Clinics

Kansas City, Missouri, United States

Site Status

Washington University/St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

Westchester Medical Center

Valhalla, New York, United States

Site Status

University of North Carolina Hospitals

Chapel Hill, North Carolina, United States

Site Status

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center - Pediatrics

Durham, North Carolina, United States

Site Status

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Oregon Health and Science University - Doernbecher Children's Hospital

Portland, Oregon, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Children's Medical Center Dallas

Dallas, Texas, United States

Site Status

Texas Transplant Institute

San Antonio, Texas, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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13-TLEC

Identifier Type: -

Identifier Source: org_study_id

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