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An Open Label,Double Arm,Single Center Pilot Study to Evaluate the Safety and Efficacy of Transplantation of Either StemEx, Umbilical Cord Blood Stem and Progenitor Cells Expanded ex Vivo, or an Unmanipulated Cord Blood Unit in the Elderly Population With Hematologic Malignancies Using Reduced Intensity Regimen

Phase 2
55 Years
73 Years
Open (Enrolling)
Hematologic Malignancies

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Trial Information

An Open Label,Double Arm,Single Center Pilot Study to Evaluate the Safety and Efficacy of Transplantation of Either StemEx, Umbilical Cord Blood Stem and Progenitor Cells Expanded ex Vivo, or an Unmanipulated Cord Blood Unit in the Elderly Population With Hematologic Malignancies Using Reduced Intensity Regimen

Allogeneic stem cell transplantation is a life saving procedure in selected high-risk or
recurrent hematologic malignancies and marrow failure syndromes. However its wide
application is limited by availability of suitably HLA matched adult donors. Umbilical Cord
Blood (UCB) has been increasingly used as an alternative hematopoietic stem cell source for
these patients. To date, over 10,000 UCB transplants have been performed in both
children32-38 and adults.35,39-44 Its advantages include easier procurement, decreased risk
to donors, reduced risk of transmitting infections, the immediate availability of
cryopreserved units, and acceptable HLA mismatches. The transplantation of UCB allows a
greater degree of HLA mismatching without an unacceptably high incidence of graft versus
host disease (GVHD). Adult patients receiving myeloablative cord blood transplants have a
90% chance of engraftment, but carry a 50% rate of transplant related mortality.

Inclusion Criteria:

- Ages 55-73

- Patients will have one of the following malignancies:

- Acute myelogenous leukemia (AML) deNovo in first CR with adverse cytogenetic
abnormalities, M0, M6, M7 subtypes, extramedullary disease in remission or high
CD34+ disease (> 50%)

- AML in early relapse (5-10% blasts on bone marrow aspirate or biopsy), or beyond
CR-1 with no circulating blasts

- AML at any time if resulting from a previous myelodysplasia

- Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL) in first CR with
adverse prognostic features: t (9; 22), extra medullary disease, or mature B
cell phenotype

- Acute lymphoid leukemia or lymphoblastic lymphoma in early relapse (5- 10%
blasts on aspirate), or beyond CR-1

- Acute Undifferentiated Leukemia or biphenotypic leukemia in CR1 or CR2

- Transfusion dependent myelodysplastic syndrome (MDS) or refractory anemia with
excess blasts (RAEB) or RAEB-in transition, CMMOL, or any myelodysplasia with
7q-, 5q-, 7-, 5- or resulting from prior anti cancer therapy.

- Relapsed Non-Hodgkin's Lymphoma (NHL), including those that have relapsed after
an autologous marrow/blood stem cell transplant

- Chronic lymphocytic leukemia (CLL) patient who has had fludarabine and either
failed or relapsed. Prior autologous transplant patients are eligible.

- Patients with adequate organ function and performance status criteria

- Subject must have at least one or the following back-up stem cell sources in case of
engraftment failure:

- Subject is willing to undergo BM harvest or peripheral blood progenitor cells
(PBPC) collection for use in case of engraftment failure (when clinically

- Subject has a second CBU as a possible back up.

- Subject's haploidentical family member has been identified and agreed (by
signing a written informed consent) to donate hematopoietic stem cells in case
of engraftment failure.

- Evaluation by social service/psychologist

- Subject signs the written informed consent after being aware of the nature of the
subject's disease and willingly consents to the treatment program after being
informed of alternative treatments, potential risks, benefits and discomforts.

- Ability to understand and agree to compliance with strict evaluation, isolation,and
medication schedules

- Designated primary care giver.

- Dental evaluation/treatment completed.

- ENT evaluation/treatment completed.

- All patient who survive to day 90 are eligible for measurement of T and B cell
function and lymphocyte subset numbers to determine immune reconstitution post UCB
transplantation with or without StemEx®

Exclusion Criteria:

- Patient with suitable related donor as defined per institutional guidelines

- Chemotherapy resistant or active AML, ALL, AUL, biphenotypic leukemia

- AML evolved from myelofibrosis

- MDS with 20% or greater bone marrow blasts at pre-transplant workup. Patients may
receive therapy and if in remission, are eligible

- Prior allogeneic hematopoeitic stem cell transplant at any time

- Less than twenty-one days have elapsed since the subject's last radiation or
chemotherapy prior to conditioning (except for hydroxyurea)

- Uncontrolled bacterial, fungal or viral infection at the time of study enrollment

- Seropositive or NAT positive for HIV, HTLV-1 and Hepatitis C

- Subjects with signs and symptoms of active central nervous system (CNS) disease

- Females who are pregnant or breastfeeding

- Allergy to bovine proteins or to aminoglycoside antibiotics (e.g. gentamicin) or to
any product, which may interfere with the treatment.

- Patient unable to give informed consent or unable to comply with the treatment
protocol including appropriate supportive care, follow-up and research tests.

- Enrolled in another clinical trial or received an investigational treatment during
the last 30 days, unless approved by the primary investigator.

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:

Efficacy of StemEx

Outcome Description:

The primary endpoint is to demonstrate the efficacy of Stemex® vs. unmanipulated UCB transplantation in the elderly population (>55years of age) following RIC regimen by demonstrating engraftment with full donor chimerism (>98%) by Day 100 of more than 60% of the patients who received transplants expanded by the Stemex method.

Outcome Time Frame:

100 days

Safety Issue:


Principal Investigator

Patrick Stiff, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Loyola Universtiy Medical Center


United States: Food and Drug Administration

Study ID:




Start Date:

January 2010

Completion Date:

January 2015

Related Keywords:

  • Hematologic Malignancies
  • Stem cell transplantation
  • Stem Cell expansion
  • Elderly
  • Neoplasms
  • Hematologic Neoplasms



Loyola University Medical Center Maywood, Illinois  60153