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Completed

NCT Number: NCT03904134

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

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.

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Key information

Conditions

Acute Myeloid Leukemia Acquired Aplastic Anemia Acute Lymphoblastic Leukemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Bone Marrow Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hodgkin Disease Hodgkin Lymphoma Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Leukemia, Myeloid Leukemia, Myeloid, Acute Lymphatic Diseases Lymphoma Lymphoma, Non-Hodgkin Lymphoproliferative Disorders Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type Non-Hodgkin Lymphoma Precursor Cell Lymphoblastic Leukemia-Lymphoma Sickle Cell Disease

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

City of Hope, Duarte, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients fulfilling the inclusion criteria will be eligible for enrollment in this study. Of those who consent, only patients who lack a suitable HLA-identical or 1 allele or antigen mismatched related donors are evaluable. Patients with an HLA-identical sibling or 1 allele or antigen mismatched family member donor are evaluable as long as the center deems the family member donor as unsuitable for other reasons. Patients may co-enroll with other interventional or observational studies.

  • Patients of all ages with AML, ALL, MDS, NHL, HL, AA, or SCD are eligible.
  • Any planned conditioning regimen and GVHD prophylaxis approach is eligible.
  • 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.
  • Patient and physician must intend to proceed with allogeneic HCT within the next 6 months if a suitable donor is identified.
  • 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.
  • 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

  • Prior allogeneic HCT (prior autologous transplant is allowed)
  • Previous formal unrelated donor search

Treatment and study plan

Donor Search Prognosis Score

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.

Primary outcomes

  1. Overall Survival for MUD Very Likely and MUD Very Unlikely Arms

    Time frame: 2 years

    Compare overall survival between Very Likely to find a matched unrelated donor search prognosis patients and Very Unlikely to find a matched unrelated donor search prognosis patients who are evaluable.

Secondary outcomes

  1. Cumulative Incidence of Transplant by Donor Search Prognosis Score

    Time frame: 2 years

    To estimate and compare the cumulative incidence of receiving a transplant according to donor search prognosis, regardless of donor search prognosis

  2. Barriers to Transplant

    Time frame: 2 years

    To describe barriers to achieving transplantation with different donor search strategies, regardless of donor search prognosis

Other outcomes

  1. Overall survival in patients transplanted for malignant diseases

    Time frame: 2 years

    To compare overall survival in patients transplanted for malignant diseases, according to the donor search prognosis and the alternative donor used.

  2. Relapse in patients transplanted for malignant diseases

    Time frame: 2 years

    To compare relapse in patients transplanted for malignant diseases, according to the donor search prognosis and the alternative donor used.

  3. Disease-free survival in patients transplanted for malignant diseases

    Time frame: 2 years

    To compare disease-free survival, treatment-related mortality, and acute and chronic GVHD in patients transplanted for malignant diseases, according to the donor search prognosis and the alternative donor used.

  4. Treatment-related mortality in patients transplanted for malignant diseases

    Time frame: 2 years

    To compare treatment-related mortality in patients transplanted for malignant diseases, according to the donor search prognosis and the alternative donor used.

  5. Acute and chronic GVHD in patients transplanted for malignant diseases

    Time frame: 2 years

    To compare acute and chronic GVHD in patients transplanted for malignant diseases, according to the donor search prognosis and the alternative donor used.

  6. Survival in patients with acquired aplastic anemia and sickle cell disease after transplantation

    Time frame: 2 years

    To describe survival in patients with acquired aplastic anemia and sickle cell disease after transplantation, according to the donor search prognosis and the alternative donor used.

  7. Acute and chronic GVHD in patients with acquired aplastic anemia and sickle cell disease after transplantation

    Time frame: 2 years

    To describe acute and chronic GVHD in patients with acquired aplastic anemia and sickle cell disease after transplantation, according to the donor search prognosis and the alternative donor used.

  8. AML or ALL in first complete remission or early stage MDS Substudy - QoL

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to compare QOL, according to the donor search prognosis and alternative donor used.

  9. AML or ALL in first complete remission or early stage MDS Substudy - primary graft failure

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of primary graft failure, according to the donor search prognosis and alternative donor used.

  10. AML or ALL in first complete remission or early stage MDS Substudy - chronic GVHD

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of chronic GVHD, according to the donor search prognosis and alternative donor used.

  11. AML or ALL in first complete remission or early stage MDS Substudy - time until off systemic immunosuppression

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe time until off systemic immunosuppression, according to the donor search prognosis and alternative donor used.

  12. AML or ALL in first complete remission or early stage MDS Substudy - GRFS

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of acute grade III-IV and chronic GVHD requiring immunosuppression-free, relapse-free survival (GRFS), according to the donor search prognosis and alternative donor used.

  13. AML or ALL in first complete remission or early stage MDS Substudy - CRFS

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of moderate-severe chronic GVHD relapse-free survival (CRFS), according to the donor search prognosis and alternative donor used.

  14. AML or ALL in first complete remission or early stage MDS Substudy - current CRFS

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of current CRFS (still on systemic treatment for cGVHD), according to the donor search prognosis and alternative donor used.

  15. QOL Substudy - number of hospital days

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the number of hospital days in the first 100 post-transplant days, according to the donor search prognosis and alternative donor used.

  16. QOL Substudy - infections

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of infections, according to the donor search prognosis and alternative donor used.

  17. QOL Substudy - immune reconstitution

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the incidence of immune reconstitution, according to the donor search prognosis and alternative donor used.

  18. QOL Substudy - late effects after transplantation

    Time frame: 2 years

    In patients with AML or ALL in first complete remission or early stage MDS treated with a limited subset of conditioning and GVHD prophylaxis regimens and transplanted with either matched unrelated donors or haploidentical related donors (QOL Substudy), to describe the late effects after transplantation, according to the donor search prognosis and alternative donor used.

Sponsors and collaborators

Lead sponsor

Center for International Blood and Marrow Transplant Research

Network

Collaborators

  • Blood and Marrow Transplant Clinical Trials Network
  • Medical College of Wisconsin
  • National Cancer Institute (NCI)
  • National Heart, Lung, and Blood Institute (NHLBI)
  • National Marrow Donor Program

Registry information

Acronym: BMT CTN 1702

Important dates

Study start
2019
Primary completion
2025
Study completion
2025
First posted
Apr 5, 2019
Registry last updated
Jan 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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