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HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies

Phase 2
24 Months
Open (Enrolling)
Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Myelodysplasia, Chronic Myeloid Leukemia, Histiocytosis

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Trial Information

HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies

Secondary objectives for this study include the following:

- To estimate the incidence of three transplant-related adverse outcomes (i.e.,
regimen-related mortality, engraftment failure, and fatal acute GVHD) in the first 100
days after transplantation.

- To estimate the incidence of chronic graft-versus-host disease.

- To evaluate those factors that affect one-year survival.

- To assess the kinetics of lymphohematopoietic reconstitution.

- To assess the frequency and clinical relevance of minimal residual disease (MRD) before
and after transplantation.

- To evaluate the incidence of and risk factors for long-term neurocognitive deficit and
organ dysfunction.

Inclusion Criteria:

Must have one of the following diagnosis:

- AML in remission or relapse (e.g., FAB M7 or biphenotypic leukemia)

- High-risk ALL in first remission (e.g., poor responder to prednisone, Ph+ ALL)

- ALL beyond first remission

- Secondary leukemia

- Primary myelodysplasia (including RAEB, RAEB-T, CMML, JCML, and JMML)

- Chronic myeloid leukemia

- Histiocytoses (including multi-system Langerhans' cell histiocytosis and
hemophagocytic lymphohistiocytosis

Inclusion criteria Donor research participants

- HIV negative (date).

- Hepatitis B surface antigen negative (date).

- Hepatitis C antibody negative (date).

- Syphilis negative (date).

- Donor is equal to or greater than 3 on 6 HLA match (date).

- Not pregnant (negative pregnancy test).

- Not lactating.

- At least 18 years of age.

Exclusion Criteria

- Patients greater than 24 months of age at the time of transplant.

- HLA-identical sibling donor is available.

- Cardiac function: shortening fraction <25%.

- Pulse oximetry oxygen saturation <92% on room air.

- Glomerular filtration rate less than 40 ml/min/1.73 m2 (may use Technetium-99 result
for GFR).

- Direct bilirubin > 3 mg/dl.

- SGPT > 500 U/L.

- Patients with previous allergy to mouse proteins.

- Patients with previous allergy to rabbit serum products.

- Patients with Down's syndrome

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To evaluate the one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a non-TBI based preparative regimen and T-lymphocyte depleted graft with a subsequent infusion of donor NK cells.

Outcome Time Frame:

5 Years

Safety Issue:


Principal Investigator

Wing H. Leung, M.D., PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

St. Jude Children's Research Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

May 2004

Completion Date:

May 2014

Related Keywords:

  • Acute Myeloid Leukemia
  • Acute Lymphocytic Leukemia
  • Myelodysplasia
  • Chronic Myeloid Leukemia
  • Histiocytosis
  • Stem cell transplantation
  • Stem cell transplant
  • Haploidentical transplant
  • Histiocytosis
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms



St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794