Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)

NCT ID: NCT03904134

Last Updated: 2026-01-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 1753 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-06-14
• Study Completion Date: 2025-01-06

Brief Summary

The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.

Detailed Description

This is a multicenter, interventional and observational study to understand factors affecting the likelihood of transplantation in patients without a human leukocyte antigen (HLA) matched family donor and to compare outcomes associated with pursuing an HLA-identical unrelated versus other alternative donor graft sources. Alternative donors are defined as any donor other than an HLA-matched or 1 antigen-mismatched related donor. Patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndromes (MDS), Non-Hodgkin lymphoma (NHL), Hodgkin lymphoma (HL), acquired aplastic anemia (AA) or sickle cell disease (SCD) are eligible. The primary comparison for the interventional study will be between two arms based on biologic assignment, analyzed on an intention-to-treat basis: Arm 1: Patients who are Very Likely to find a matched unrelated donor (MUD), defined as having a >90% chance of finding an 8/8 HLA-matched unrelated donor, for whom a fully matched unrelated donor will be pursued; and Arm 2: Patients who are Very Unlikely to find a MUD, defined as having a <10% chance of finding an 8/8 HLA-matched unrelated donor, for whom a haploidentical, cord blood, or mismatched unrelated donor transplant will be pursued. Patients with a Less Likely chance of finding a MUD, i.e., those not falling into the other two groups (a 26% chance), will be enrolled onto the observational component of the study and analyzed for all relevant endpoints but will not be included in the primary comparison.

Conditions

Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes; Non-hodgkin Lymphoma; Hodgkin Lymphoma; Acquired Aplastic Anemia; Sickle Cell Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Donor Search Prognosis: MUD Very Likely

Patients who are Very Likely to find a matched unrelated donor (MUD), defined as having a \>90% chance of finding an 8/8 HLA-matched unrelated donor, for whom a fully matched unrelated donor will be pursued.

Group Type: OTHER

Donor Search Prognosis Score

Intervention Type: OTHER

Patients will be placed on a study arm after receiving a Donor Search Prognosis Score, which is based on HLA allele frequencies and race/ethnicity. This score predicts the likelihood of successfully identifying a 10/10 matched unrelated donor.Worse search prognosis is associated with racial and ethnic minority status but not with other patient and disease biology characteristics that might influence the success of hematopoietic cell transplantation (HCT). Thus, the use of donor search prognosis in this trial as a tool for biologic assignment to matched unrelated donors vs. mismatched donors provides a mechanism to minimize bias from disease characteristics.

Donor Search Prognosis: MUD Very Unlikely

Patients who are Very Unlikely to find a MUD, defined as having a \<10% chance of finding an 8/8 HLA-matched unrelated donor, for whom a haploidentical, cord blood, or mismatched unrelated donor transplant will be pursued.

Group Type: OTHER

Donor Search Prognosis Score

Intervention Type: OTHER

Patients will be placed on a study arm after receiving a Donor Search Prognosis Score, which is based on HLA allele frequencies and race/ethnicity. This score predicts the likelihood of successfully identifying a 10/10 matched unrelated donor.Worse search prognosis is associated with racial and ethnic minority status but not with other patient and disease biology characteristics that might influence the success of hematopoietic cell transplantation (HCT). Thus, the use of donor search prognosis in this trial as a tool for biologic assignment to matched unrelated donors vs. mismatched donors provides a mechanism to minimize bias from disease characteristics.

Donor Search Prognosis: MUD Less Likely

Patients with a Less Likely chance of finding a MUD, i.e., those not falling into the other two groups (a 26% chance), will be enrolled onto the observational component of the study and analyzed for all relevant endpoints but will not be included in the primary comparison.

Group Type: OTHER

Donor Search Prognosis Score

Intervention Type: OTHER

Patients will be placed on a study arm after receiving a Donor Search Prognosis Score, which is based on HLA allele frequencies and race/ethnicity. This score predicts the likelihood of successfully identifying a 10/10 matched unrelated donor.Worse search prognosis is associated with racial and ethnic minority status but not with other patient and disease biology characteristics that might influence the success of hematopoietic cell transplantation (HCT). Thus, the use of donor search prognosis in this trial as a tool for biologic assignment to matched unrelated donors vs. mismatched donors provides a mechanism to minimize bias from disease characteristics.

Interventions

Donor Search Prognosis Score

Patients will be placed on a study arm after receiving a Donor Search Prognosis Score, which is based on HLA allele frequencies and race/ethnicity. This score predicts the likelihood of successfully identifying a 10/10 matched unrelated donor.Worse search prognosis is associated with racial and ethnic minority status but not with other patient and disease biology characteristics that might influence the success of hematopoietic cell transplantation (HCT). Thus, the use of donor search prognosis in this trial as a tool for biologic assignment to matched unrelated donors vs. mismatched donors provides a mechanism to minimize bias from disease characteristics.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Patients of all ages with AML, ALL, MDS, NHL, HL, AA, or SCD are eligible.

2. Any planned conditioning regimen and GVHD prophylaxis approach is eligible.

3. Patients must be considered suitable allogeneic transplant candidates at the time of enrollment based on medical history, physical examination, and available laboratory tests. Specific testing for organ function is not required for eligibility but, if available, these tests should be used by the treating physician to judge transplant suitability.

4. Patient and physician must intend to proceed with allogeneic HCT within the next 6 months if a suitable donor is identified.

5. Center plans to follow the algorithm for alternative donor identification: (a) for subjects who are Very Likely to find a MUD, attempt to identify a matched unrelated donor; (b) for a subjects who are Very Unlikely to find a MUD, proceed expeditiously to a haploidentical, cord blood or mismatched unrelated donor.

6. Signed informed consent, and assent if applicable. Consent may be signed prior to completion of family typing but patients will only be considered evaluable upon confirmation that there is no suitable HLA-identical or 1 allele or antigen mismatched related donor available.

Exclusion Criteria

1. Prior allogeneic HCT (prior autologous transplant is allowed)

2. Previous formal unrelated donor search

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Blood and Marrow Transplant Clinical Trials Network

NETWORK

Sponsor Role: collaborator

National Marrow Donor Program

OTHER

Sponsor Role: collaborator

Medical College of Wisconsin

OTHER

Sponsor Role: collaborator

Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Stephanie J Lee, MD, MPH

Role: STUDY_CHAIR

Fred Hutchinson Cancer Center

Stefan Ciurea, MD

Role: STUDY_CHAIR

M.D. Anderson Cancer Center

Locations

City of Hope

Duarte, California, United States

University of California, San Diego Medical Center

La Jolla, California, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Stanford Hospitals and Clinics

Stanford, California, United States

Children's National Medical Center

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

University of Florida

Gainesville, Florida, United States

University of Miami

Miami, Florida, United States

Memorial Healthcare System

Pembroke Pines, Florida, United States

H. Lee Moffitt Cancer Center

Tampa, Florida, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Emory University

Atlanta, Georgia, United States

Northside Hospital

Atlanta, Georgia, United States

Loyola University

Maywood, Illinois, United States

Indiana University

Indianapolis, Indiana, United States

University of Maryland

Baltimore, Maryland, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

University of Michigan

Ann Arbor, Michigan, United States

Karmanos Cancer Institute

Detroit, Michigan, United States

University of Minnesota

Minneapolis, Minnesota, United States

Mayo Clinic Rochester

Rochester, Minnesota, United States

University of Mississippi

Jackson, Mississippi, United States

Children's Mercy Hospital

Kansas City, Missouri, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

Washington University in St. Louis

St Louis, Missouri, United States

University of Nebraska Medical Center - Adults

Omaha, Nebraska, United States

University of Nebraska Medical Center - Pediatrics

Omaha, Nebraska, United States

Rosewell Park Cancer Institute

Buffalo, New York, United States

Mount Sinai Medical Center

New York, New York, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Levine Cancer Institute

Charlotte, North Carolina, United States

Duke University Medical Center

Durham, North Carolina, United States

Wake Forest Baptist Health

Winston-Salem, North Carolina, United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Cleveland Clinic

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

The Ohio State University

Columbus, Ohio, United States

University of Oklahoma

Oklahoma City, Oklahoma, United States

Oregon Health and Science University

Portland, Oregon, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Children's Medical Center Dallas

Dallas, Texas, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

M.D. Anderson Cancer Center

Houston, Texas, United States

University of Utah

Salt Lake City, Utah, United States

University of Virginia

Charlottesville, Virginia, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

University of Wisconsin

Madison, Wisconsin, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

5U24HL138660-02

Identifier Type: NIH

Identifier Source: secondary_id

View Link

N00014-18-1-2888

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

BMT CTN 1702

Identifier Type: -

Identifier Source: org_study_id