HAPNK1: Blood and Marrow Transplant (BMT) for Children with Leukemia/Lymphoma

Haploidentical Donor Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation with a TLI-Based Conditioning Regimen in Patients with Hematologic Malignancies

Category:

Bone Marrow Transplant

Diseases Treated:

Leukemia/Lymphoma

Eligibility Overview:

  • 21 years and younger
  • Does not have a suitable HLA-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate for conventional matched donor transplant due to refractory disease
  • Has a suitable single haplotype-matched family member donor
  • High-risk hematologic malignancy, including certain diagnoses of:
    • Acute lymphoblastic leukemia (ALL)
    • Acute myeloid leukemia (AML)
    • Chronic myelogenous leukemia (CML)
    • Myelodysplastic syndrome (MDS)
    • Hodgkin lymphoma
    • Non-Hodgkin lymphoma
  1. Brief Summary

    Studies at St. Jude Children’s Research Hospital and other institutions have demonstrated that hematopoietic cell transplantation (HCT) is a potential curative therapy for certain aggressive hematologic malignancies, including chronic myelogenous leukemia, acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndrome. Despite these findings, approximately 25 to 60% of children who are transplant candidates will not have an available human leukocyte antigen (HLA)-matched related/sibling donor or an HLA-matched unrelated donor. Yet, nearly all patients will have a readily available haploidentical donor.

    Relapse, infection and graft versus host disease (GVHD) are major concerns after HCT. However, recent studies suggest that alloreactive natural killer (NK) cells may help reduce GVHD, graft rejection and viral infections as well as lessen the chance of relapse. In addition, donor memory T cells provide potent viral protection and may also lessen the chance of relapse. Therefore, this clinical trial will evaluate the effectiveness of haploidentical donor allogeneic HCT which is selectively T-cell depleted such that donor memory cells remain available for infusion, along with the addition of donor NK cells, in this high-risk patient population.

    Primary Objective

    • To estimate the rate of successful engraftment at day +42 post-transplant in patients who receive selectively T-cell depleted haploidentical donor progenitor cell plus NK cell transplantation with TLI-based conditioning regimen for high-risk hematologic malignancy

    Eligibility Criteria

    Inclusion criteria for transplant recipient include:

    • 21 years and younger
    • Does not have a suitable HLA-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate due to refractory disease
    • Has a suitable single haplotype-matched  family member donor
    • High-risk hematologic malignancy, including certain diagnoses of:
      • Acute lymphoblastic leukemia (ALL)
      • Acute myeloid leukemia (AML)
      • Chronic myelogenous leukemia (CML)
      • Myelodysplastic syndrome (MDS)
      • Hodgkin lymphoma
      • Non-Hodgkin lymphoma

    Inclusion criteria for haploidentical donor include:

    • At least single haplotype matched (≥ 3 of 6) family member
    • 18 years of age and older
    • HIV negative
    • Not pregnant

    Study Sites

    St. Jude Children’s Research Hospital
    Memphis, Tennessee

  2. About this study

    Hematologic cancers are cancers of the blood and lymphatic system. These include leukemias and lymphomas. Generally, patients with blood cancers are treated successfully with strong anti-cancer medicines, also known as chemotherapy. However, some blood cancers can be hard to treat. They may come back after treatment or may respond poorly to treatment.

    This clinical trial will study allogeneic hematopoietic cell transplant (sometimes called bone marrow transplant) as a treatment for these difficult cancers. Allogeneic transplantation uses hematopoietic progenitor (blood making) cells that are obtained from someone other than the patient. That person is called the donor. These cells are given to the patient in a procedure called infusion.

    Standard transplants begin with radiation and/or chemotherapy to damage or kill your bone marrow. Bone marrow is mostly located in the large bones of the body like the hips and leg bones. Killing or damaging the bone marrow makes room within the bones for cells donated by someone else. The body needs these donor progenitor (blood making) cells to restore and rebuild bone marrow. The donor cells travel through the patient’s blood to the bone marrow space and begin to grow and make new blood cells. Immune system cells from the donor are also infused. These immune cells can sometimes attack the cancer cells and prevent them from coming back.

    Purpose of this clinical trial

    The main purpose of this study is to see how well this type of transplant works in children and adolescents with certain kinds of blood cancers that have come back after treatment or not responded well to treatment. Researchers want to know the good and bad effects of the transplant. They also want to learn more about how the body’s immune system responds to the transplant.

    Eligibility overview

    • 21 years and younger
    • Does not have a suitable human leukocyte antigen (HLA)-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate due to refractory disease
    • Has a suitable single haplotype-matched  family member donor
    • High-risk hematologic malignancy, including certain diagnoses of:
      • Acute lymphoblastic leukemia (ALL)
      • Acute myeloid leukemia (AML)
      • Chronic myelogenous leukemia (CML)
      • Myelodysplastic syndrome (MDS)
      • Hodgkin lymphoma
      • Non-Hodgkin lymphoma
  3. HAPNK1 Quick View
    Sponsors St. Jude Children’s Research Hospital
    ClinicalTrials.gov identifier NCT01807611
    Trial Start Date April 2013
    Estimated Enrollment 110
    Study Type Interventional
    Study Phase Phase II
    Conditions Leukemia, lymphoma
    Ages 21 years and younger (recipient)
    Principal investigator Brandon M. Triplett, MD
    Study Sites St. Jude Children’s Research Hospital
    For a consultation or to discuss HAPNK1 St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

Contact

Brandon M. Triplett, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.