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Biparental HLA Haplotype Disparate T-cell Depleted Transplants for Patients Lacking an HLACompatible Donor

Phase 2
19 Years
Open (Enrolling)
Acute Leukemia, Chronic Leukemia, Myelodysplastic Syndrome, Non-Hodgkins Lymphoma

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Trial Information

Biparental HLA Haplotype Disparate T-cell Depleted Transplants for Patients Lacking an HLACompatible Donor

Inclusion Criteria:

- Malignant conditions for which CD34+ selected, T-cell depleted allogeneic
hematopoietic stem cell transplantation is indicated such as:

AML in 1st remission - for patients whose AML does not have 'good risk' cytogenetic
features (i.e. t 8;21, t15;17, inv 16).

- Secondary AML in 1st remission

- AML in 1st relapse or > 2nd remission

- ALL/LL in 1st remission clinical or molecular features indicating a high risk for
relapse; or ALL > 2nd remission

- CML failing to respond to or not tolerating Imatinib, dasatinib, or nilotinib in
first chronic phase of disease; or CML in accelerated phase or second chronic phase.

- Non-Hodgkins lymphoma with chemoresponsive disease in any of the following
categories: a) intermediate or high grade lymphomas who have failed to achieve a
first CR or have relapsed following a 1st remission who are not candidates for
autologous transplants.

- any NHL in remission which is considered not curable with chemotherapy alone and not
eligible/appropriate for autologous transplant. Myelodysplastic syndrome (MDS):
RA/RCMD with high risk cytogenetic features or transfusion dependence, RAEB-1 and
RAEB-2 and Acute myelogenous leukemia (AML) evolved from MDS, who are not eligible
for transplantation under protocol IRB 08-008.

- Chronic myelomonocytic leukemia: CMML-1 and CMML-2.

- Other rare lethal disorders of Hematopoiesis and Lymphopoiesis for which a T-cell
depleted transplant is indicated (e.g. hemophagocytic lymphohistiocytosis; refractory
aplastic anemia or conjugated cytopenias; non-SCID lethal genetic immunodeficiencies
such as Wiskott Aldrich Syndrome, CD40 ligand deficiency, ALPS).

- Patients may be of either gender and of any racial or ethnic background.

- Patients must have a Karnofsky (adult) or Lansky (pediatric) Performance Status >

- Patients must have adequate organ function measured by:

Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > 50% and must improve
with exercise.

- Hepatic: < 3x ULN ALT and < 2.0x ULN total serum bilirubin, unless there is
congenital benign hyperbilirubinemia.

- Renal: serum creatinine <1.2 mg/dl or if serum creatinine is outside the normal
range, then CrCl > 40 ml/min (measured or calculated/estimated)

- Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for

- Each patient must be willing to participate as a research subject and must sign an
informed consent form.

Exclusion Criteria:

- Female patients who are pregnant or breast-feeding

- Uncontrolled viral, bacterial or fungal infection

- Patient seropositive for HIV-I/II; HTLV -I/II

- Presence of leukemia in the CNS.


Inclusion Criteria:

- Two donors will be required for each transplant. Each HLA -A, B, C, DR, DQ
genotypically haplotype disparate related donor, should inherit one of the two HLA
haplotypes inherited by the patient. The second related donor should inherit the
patient's other HLA haplotype.

- Each donor must meet criteria outlined in the FACT-approved SOP for "DONOR EVALUATION
Program Manual, document E-1 (see attached, or link to URL:

- Donor must have adequate peripheral venous catheter access for leukapheresis or must
agree to placement of a central catheter.

- Wt >25kg

Donor Exclusion Criteria:

- Evidence of active infection (including urinary tract infection, or upper respiratory
tract infection), viral hepatitis exposure (on screening), unless only HBS Ab+ and
HBV DNA negative, or serologic evidence of exposure or infection with HIV-I/II or

- Medical or physical reason which makes the donor unlikely to tolerate or cooperate
with growth factor therapy and leukapheresis

- Factors which place the donor at increased risk for complications from leukapheresis
or G-CSF therapy (e.g., autoimmune disease, sickle cell trait, symptomatic coronary
artery disease requiring therapy).

- Pregnancy (positive serum or urine β-HCG) or breastfeeding. Women of childbearing age
must avoid becoming pregnant while on the study

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:


Outcome Description:

measured by: incidence of transplant-related mortality, overall survival and disease-free survival at 1 year post transplant. incidence, tempo and complications of engraftment and hematopoietic reconstitutions and conversely, the risk of graft failure incidence and severity of acute and/or chronic GVHD incidence and severity of opportunistic infections developing following engraftment

Outcome Time Frame:

1 year

Safety Issue:


Principal Investigator

Richard O'Reilly, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Memorial Sloan-Kettering Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

May 2012

Completion Date:

May 2014

Related Keywords:

  • Acute Leukemia
  • Chronic Leukemia
  • Myelodysplastic Syndrome
  • Non-Hodgkins Lymphoma
  • CliniMACS-CD34 Reagent System
  • 12-053
  • Leukemia
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Myelodysplastic Syndromes
  • Preleukemia
  • Chronic Disease



Memorial Sloan-Kettering Cancer CenterNew York, New York  10021