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Myeloablative Allogeneic Stem Cell Transplantation Using a Naive T-Cell Depleted Peripheral Blood Stem Cell Graft

Phase 1/Phase 2
18 Years
65 Years
Open (Enrolling)

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Trial Information

Myeloablative Allogeneic Stem Cell Transplantation Using a Naive T-Cell Depleted Peripheral Blood Stem Cell Graft

A cohort of patients (Cohort 1) will be enrolled to receive the currently accepted standard
approach to myeloablative allogeneic stem cell transplantation. With the exception of
volume and/or plasma depletion (in cases of donor/recipient ABO incompatibility), the
peripheral blood stem cell graft will be unmodified. The primary purpose of Cohort 1 is to
prospectively collect samples for measurement of immune recovery from a relatively
homogeneous population of patients treated in a uniform manner. Within the limitations of
age-matching, patients accrued to Cohort 1 will be incorporated into a larger retrospective
historical control group for purposes of comparison with Cohort 2 of the incidence of grade
II-IV acute Graft versus Host disease. The experimental aspects of this trial will be the
use of a naïve T-cell depleted peripheral blood stem cell graft (Cohort 2). All other
aspects of this stem cell transplantation are in line with the standard of care.
Recruitment to this trial will be stratified by donor type as matched sibling or matched
unrelated donor. Patients will be conditioned with total body irradiation (1350cGy) and
Cyclophosphamide. The donor stem cell grafts will come from mobilized peripheral blood of
6/6 HLA-identical family members or 8/8 (HLA A, B, C, DRB1) allele-level matched unrelated

Inclusion Criteria:

- Age 18 to 65 years.

- 8/8 or 7/8 HLA-identical matched sibling OR Allele level 8/8 (HLA-A, B, C, DRbeta1)
matched unrelated donor.

- Patients with high risk ALL in first complete remission, with high risk being defined
by the presence of t(4;11), t(9;22) or t(1;19) or patients presenting with extreme
hyperleukocytosis (WBC>500,000/ml) or partial remission after initial induction

- Adult patients with acute non-lymphocytic Leukemia (ANLL) in first complete remission
with high-risk cytogenetics (monosomy chromosome 5 or 7, del(5q), abn(3q26), complex
karyotypic abnormalities) or failure to achieve complete remission after standard
induction therapy.

- All patients with ALL or ANLL in second or subsequent remission or partial remission
(<5% blasts in bone marrow as measured by flow cytometry).

- All patients with CML in chronic (failed interferon and/or Gleevec) or accelerated

- Patients with myelodysplastic syndrome with International Prognostic Scoring System
(IPSS) risk category of INT-1 or greater.

- Myelofibrosis with myeloid metaplasia

- Patients with severe aplastic anemia must have failed immunosuppressive therapy such
as cyclosporine plus anti-thymocyte globulin.

- Patients with a history of CNS disease must have been treated and have no active CNS
disease at the time of protocol treatment.

- ECOG performance status <2

- Patients must have adequate function of other organ systems as measured by:

- Creatinine clearance (by Cockcroft Gault equation [Appendix IV]) > 30ml/min. Hepatic
transaminases (ALT/AST) < 4 x normal, bilirubin < 2.0 mg/dl.

- Pulmonary function tests demonstrating FVC and FEV1 of >50% of predicted for age and
DLCO > 50% of predicted.

- Ejection fraction of >45% by echocardiogram, radionuclide scan or cardiac MRI.

- Patients must be HIV negative.

- Patients must not be pregnant.

Exclusion Criteria:

- Patients with > 5% blasts in bone marrow or peripheral circulation.

- Patients with rapidly progressive ANLL or ALL.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The incidence of grade II/IV acute graft versus host disease and one year disease-free survival.

Outcome Time Frame:

One year

Safety Issue:


Principal Investigator

Mitchell Horwitz, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University Health System


United States: Food and Drug Administration

Study ID:




Start Date:

July 2007

Completion Date:

July 2017

Related Keywords:

  • ALL
  • ANLL
  • MDS
  • NHL
  • myeloablative
  • stem cell
  • naive T-cell depleted
  • allogeneic
  • Leukemia, Myeloid, Acute