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Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options

Phase 2
18 Years
65 Years
Not Enrolling
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Blastic Phase Chronic Myelogenous Leukemia, Cutaneous B-cell Non-Hodgkin Lymphoma, de Novo Myelodysplastic Syndromes, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Intraocular Lymphoma, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Noncutaneous Extranodal Lymphoma, Peripheral T-cell Lymphoma, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Small Intestine Lymphoma, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Testicular Lymphoma, Waldenström Macroglobulinemia

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Trial Information

Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options


I. To determine the engraftment parameters (time to absolute neutrophils > 500/mm^3) and 100
day non-relapse mortality after umbilical cord blood (UCB) transplantation under the
following conditions: a) Double UCB unit transplantation (Arm 1 of this study); b) Single
UCB unit transplantation in conjunction with irradiated haploidentical peripheral blood
mononuclear cells (PBMCs) (Arm 2 of this study).


I. To determine transplant-related mortality (1 year overall and malignancy-free survival),
incidence and staging of graft-versus-host disease (GVHD), rate of infection, and rate of
immune reconstitution under the two transplant conditions.

OUTLINE: Patients are assigned to 1 of 2 treatment arms.

ALL PATIENTS: Patients receive conditioning comprising fludarabine phosphate intravenously
(IV) over 30 minutes on days -6 to -2, cyclophosphamide IV over 1 hour on day -6, and
undergo total-body irradiation (TBI) on day -1. Patients also receive GVHD prophylaxis
comprising tacrolimus IV continuously or orally (PO) beginning on day -3 with taper and
mycophenolate mofetil PO twice daily (BID) days 1-30.

ARM I: Patients undergo double allogeneic UCB transplant on day 0.

ARM II: Patients undergo single allogeneic UCB transplant on day 0. Patients also undergo
irradiated allogeneic PBMC transplant within 8 hours following the UCB infusion.

Treatment continues in the absence of disease progression or unacceptable toxicity.

Inclusion Criteria:

- Patients with histologically proven hematologic malignancy with anticipated 2 year
survival < 20% with standard therapy; patients age <18 are excluded by virtue of the
policies and procedures of the allogeneic hematopoietic stem cell transplant (HSCT)
program (Cancer Institute of New Jersey [CINJ]/Robert Wood Johnson University
Hospital [RWJUH] is not an approved Pediatric Transplant Center); patients > age 65
are generally not considered candidates for experimental unrelated allogeneic HSCT,
as utilized in this study by virtue of the anticipated delayed immune reconstitution,
high risk of GVHD, and known negative impact of age on outcomes

- Patients eligible for this trial will have high risk diseases that include, but are
not limited to:

- Acute myeloid leukemia (AML) in second complete remission (CR2) or greater or
early relapse with < 5% marrow blasts and no circulating blasts

- AML in first complete remission (CR1) with high risk cytogenetics (complex,
monosomy 5, monosomy 7, 11q23 (not t(9;11)), t(6;9), chromosome 3, monosomy
phenotype and other karyotypes estimated to have =< 20% disease free survival at
3 years) or secondary/transformed AML without favorable cytogenetics;

- Acute lymphoblastic leukemia (ALL) with t(9;22), 11q23 abnormality or early
relapse (< 5% marrow blasts) or CR2 or greater;

- Chronic myeloid leukemia (CML) resistant/refractory to all commercially
available Abelson (abl) kinase inhibitors (e.g. imatinib mesylate, dasatinib,
nilotinib) or predicted to be so based upon clinical course or abl kinase domain
mutation analysis; or in accelerated phase or blast crisis;

- High intermediate to high international prognostic score myelodysplasia;

- Non-Hodgkin lymphoma (NHL)/Hodgkin lymphoma (HL)/other lymphoproliferative
diseases resistant/refractory to standard therapies and for whom an autologous
transplant is considered to be inappropriate (e.g. bone marrow involvement,
chemotherapy refractory disease, prior transplant);

- Chronic lymphocytic leukemia (CLL) resistant/refractory to standard therapies
(e.g. fludarabine) or high risk cytogenetics/fluorescence in situ hybridization
(FISH) (e.g. 17p-);

- Myeloproliferative disorders with progressive disease or cytopenias or clinical
symptoms refractory to standard therapy (e.g. hypomethylating agents)

- Relapsed or refractory multiple myeloma after (or not eligible for) high dose
chemotherapy/autologous hematopoietic stem cell rescue and following salvage
therapy with thalidomide, lenalidomide or bortezomib/other Food and Drug
Administration (FDA)-approved multiple myeloma salvage therapies;

- Other hematologic malignancies/disorders with anticipated 2 year survival < 20%,
as established by available data bases, medical literature and the documented
consensus of the Hematologic Malignancies Tumor Study Group

- Patients must be an allogeneic HSCT candidate but have no standard donor (matched
related donor [MRD], human leukocyte antigen [HLA]-matched unrelated donor [MUD] or
single UCB unit of appropriate size and HLA type) available

- Patients must have available UCB unit(s)

- Patients considered for Arm 2 must not be eligible for Arm 1 and must have an
HLA-haploidentical sibling, parent, child, or other relative (uncle, aunt, first
cousin, niece or nephew) who meets donor requirements as outlined in Donor
Eligibility criteria

- Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status =<

- Left ventricular (LV) ejection fraction >= 50%

- Diffusion capacity of carbon monoxide (DLCO) corrected for hemoglobin > 60%

- Total bilirubin within normal institutional limits unless the patient has Gilbert's

- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase
[SGOT])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT])
=< 2.5 X institutional upper limit of normal (ULN)

- Measured or estimated creatinine clearance > 50 ml/min

- Hematopoietic stem cell co-morbidity index =< 2

- There must be a negative pregnancy test for women of childbearing potential within 1
week of therapy; there must be willingness to avoid pregnancy and undergo counseling
about contraceptive techniques throughout the course of treatment

- There must be no uncontrolled infections or active acute or chronic illnesses such as
diabetes, angina/myocardial ischemia, cardiac arrhythmia, venous thrombosis/embolism,
cerebrovascular disease, seizure disorder, psychiatric illness or other intercurrent
illness that is not well controlled or is anticipated to be difficult to control
during the proposed therapy

- The patient must be aware of the high risk and experimental nature of the treatment
and provide informed consent

- The patient must have clearance for HSCT after psychosocial evaluation

- The patient must have adequate insurance or other support to meet the anticipated
financial burden imposed by the costs of therapy

- DONOR (for allogeneic lymphocytes, Arm 2 only): Relative (parent, child, sibling,
first cousin, uncle aunt, nephew, niece) with appropriate HLA match (>= 3/6 HLA A, B,
DR match)

- DONOR (for allogeneic lymphocytes, Arm 2 only): Age >= 18 years old

- DONOR (for allogeneic lymphocytes, Arm 2 only): Normal hemogram; potential donors not
having a normal hemogram may be utilized at the discretion of the Principal

- DONOR (for allogeneic lymphocytes, Arm 2 only): Not pregnant or lactating

- DONOR (for allogeneic lymphocytes, Arm 2 only): Not human immunodeficiency virus
(HIV)-1, HIV-2, hepatitis C (HCV), Hepatitis B core or human T-lymphotropic virus
(HTLV)-I/II seropositive; hepatitis B surface antigen (HB Sag)(-); must meet other
infectious disease screening criteria utilized by New Brunswick Affiliated Hospital
(NBAH) Blood Center

- DONOR (for allogeneic lymphocytes, Arm 2 only): No uncontrolled infections, other
medical or psychological/social conditions, or required medications that might
increase the likelihood of patient or donor adverse effects or poor outcomes

- DONOR (for allogeneic lymphocytes, Arm 2 only): Meet other blood bank criteria for
blood product donation (as determined by NBAH Blood Center screening history)

- DONOR (for allogeneic lymphocytes, Arm 2 only): Donors must be informed of the
investigational nature of this study, understand the requirements, potential benefits
and potential risks of the experimental treatment, and give written informed consent
in accordance with institutional and federal guidelines

Exclusion Criteria:

- Prior extensive radiation therapy that the radiation oncologist feels precludes
additional TBI

- Patients with known human immunodeficiency virus (HIV) are excluded due to side
effects of the therapy on the immune system

- Patients with known active central nervous system (CNS) disease will be excluded from
this clinical trial because they often develop progressive neurologic dysfunction
unresponsive to HSCT therapy

Type of Study:


Study Design:

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

Outcome Measure:

Engraftment of white blood cells (WBC) (absolute neutrophil count > 500/mm^3)

Outcome Time Frame:

By day 42

Safety Issue:


Principal Investigator

Roger Strair

Investigator Role:

Principal Investigator

Investigator Affiliation:

Cancer Institute of New Jersey


United States: Food and Drug Administration

Study ID:




Start Date:

December 2012

Completion Date:

December 2017

Related Keywords:

  • Accelerated Phase Chronic Myelogenous Leukemia
  • Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
  • Adult Acute Lymphoblastic Leukemia in Remission
  • Adult Acute Myeloid Leukemia in Remission
  • Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
  • Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
  • Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
  • Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
  • Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
  • Adult Nasal Type Extranodal NK/T-cell Lymphoma
  • Anaplastic Large Cell Lymphoma
  • Angioimmunoblastic T-cell Lymphoma
  • Blastic Phase Chronic Myelogenous Leukemia
  • Cutaneous B-cell Non-Hodgkin Lymphoma
  • de Novo Myelodysplastic Syndromes
  • Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue
  • Hepatosplenic T-cell Lymphoma
  • Intraocular Lymphoma
  • Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable
  • Nodal Marginal Zone B-cell Lymphoma
  • Noncutaneous Extranodal Lymphoma
  • Peripheral T-cell Lymphoma
  • Post-transplant Lymphoproliferative Disorder
  • Previously Treated Myelodysplastic Syndromes
  • Recurrent Adult Acute Lymphoblastic Leukemia
  • Recurrent Adult Acute Myeloid Leukemia
  • Recurrent Adult Burkitt Lymphoma
  • Recurrent Adult Diffuse Large Cell Lymphoma
  • Recurrent Adult Diffuse Mixed Cell Lymphoma
  • Recurrent Adult Diffuse Small Cleaved Cell Lymphoma
  • Recurrent Adult Grade III Lymphomatoid Granulomatosis
  • Recurrent Adult Hodgkin Lymphoma
  • Recurrent Adult Immunoblastic Large Cell Lymphoma
  • Recurrent Adult Lymphoblastic Lymphoma
  • Recurrent Adult T-cell Leukemia/Lymphoma
  • Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma
  • Recurrent Grade 1 Follicular Lymphoma
  • Recurrent Grade 2 Follicular Lymphoma
  • Recurrent Grade 3 Follicular Lymphoma
  • Recurrent Mantle Cell Lymphoma
  • Recurrent Marginal Zone Lymphoma
  • Recurrent Mycosis Fungoides/Sezary Syndrome
  • Recurrent Small Lymphocytic Lymphoma
  • Refractory Chronic Lymphocytic Leukemia
  • Refractory Hairy Cell Leukemia
  • Refractory Multiple Myeloma
  • Relapsing Chronic Myelogenous Leukemia
  • Secondary Acute Myeloid Leukemia
  • Secondary Myelodysplastic Syndromes
  • Small Intestine Lymphoma
  • Splenic Marginal Zone Lymphoma
  • T-cell Large Granular Lymphocyte Leukemia
  • Testicular Lymphoma
  • Waldenström Macroglobulinemia
  • Congenital Abnormalities
  • Blast Crisis
  • Burkitt Lymphoma
  • Neoplasms
  • Hodgkin Disease
  • Immunoblastic Lymphadenopathy
  • Leukemia
  • Leukemia, Lymphocytic, Chronic, B-Cell
  • Leukemia, Hairy Cell
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myeloid, Accelerated Phase
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Leukemia, T-Cell
  • Leukemia-Lymphoma, Adult T-Cell
  • Lymphoma
  • Lymphoma, Follicular
  • Lymphoma, Large B-Cell, Diffuse
  • Lymphoma, Non-Hodgkin
  • Lymphomatoid Granulomatosis
  • Lymphoproliferative Disorders
  • Waldenstrom Macroglobulinemia
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Mycoses
  • Mycosis Fungoides
  • Myelodysplastic Syndromes
  • Preleukemia
  • Myeloproliferative Disorders
  • Sezary Syndrome
  • Lymphoma, B-Cell
  • Lymphoma, Large-Cell, Immunoblastic
  • Lymphoma, T-Cell
  • Lymphoma, T-Cell, Cutaneous
  • Lymphoma, T-Cell, Peripheral
  • Lymphoma, Large-Cell, Anaplastic
  • Lymphoma, B-Cell, Marginal Zone
  • Lymphoma, Extranodal NK-T-Cell
  • Lymphoma, Mantle-Cell
  • Hematologic Neoplasms
  • Myelodysplastic-Myeloproliferative Diseases
  • Leukemia, Large Granular Lymphocytic



Cancer Institute of New Jersey New Brunswick, New Jersey  08901