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A Multi-Center, Multi-National, Historical Cohort Controlled Study to Evaluate Efficacy and Safety of Transplantation of StemEx®, Umbilical Cord Blood Stem and Progenitor Cells Expanded Ex Vivo, in Subjects With Hematologic Malignancies Following Myeloablative Therapy


Phase 2/Phase 3
12 Years
55 Years
Open (Enrolling)
Both
Hematologic Malignancies, Acute Myeloid Leukemia, Lymphoid Leukemia, Chronic Myeloid Leukemia, Hodgkin's Disease, Non-Hodgkin's Lymphoma, Myelodysplastic Syndromes

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Trial Information

A Multi-Center, Multi-National, Historical Cohort Controlled Study to Evaluate Efficacy and Safety of Transplantation of StemEx®, Umbilical Cord Blood Stem and Progenitor Cells Expanded Ex Vivo, in Subjects With Hematologic Malignancies Following Myeloablative Therapy


Allogeneic hematopoietic stem cell transplantation is a life-saving procedure for patients
with hematologic malignancies; yet wide application of this procedure is limited by the
availability of suitably Human Leukocyte Antigen (HLA) - matched donors. Only 30% of
patients who could benefit from this procedure have an HLA-matched sibling. The lengthy
search for a matched donor may critically delay transplantation. In addition, far fewer
patients of racial minorities find suitable HLA-matched donors. Umbilical cord blood (UCB)
has been increasingly used as an alternative source of stem cells; however, its use in
adults and adolescent patients is limited due to insufficient cell dose required for
satisfactory hematopoietic reconstitution.

Gamida Cell - Teva Joint Venture Ltd. is engaged in the development of StemEx®, an expanded
hematopoietic UCB stem cell graft, as a potential medicinal product for the treatment of
cancer and hematological malignancies. The expansion technology enables preferential
expansion of hematopoietic stem and early progenitor cells and is based on the findings that
copper chelators can regulate the balance between self-renewal and differentiation of stem
cells.

The multi-national, multi-center Phase II/III clinical study designated to evaluate the
safety and efficacy of StemEx® will enroll approximately 100 subjects with high-risk
hematologic malignancies who are candidates for allogeneic stem cell transplantation (SCT).
This study will evaluate the effect of StemEx® on overall survival as measured by overall
100-day mortality.

The study consists of 4 phases:

1. Screening phase includes subjects' clinical assessment and screening tests

2. Conditioning phase includes the myeloablative treatment prior transplantation procedure

3. Transplantation and post-transplant follow-up phase to day 180

4. Observational phase: survival status follow-up to day 730 (18 months)


Inclusion Criteria:



1. Clinical diagnosis of AML or ALL: CR2 or subsequent complete remission (CR) or
CR1 with high-risk features or relapse with < 10% blasts in BM and no circulating
blasts.

2. Clinical diagnosis of CML: in CP1 (Chronic Phase 1) and resistant or intolerant to
Gleevec or in CP2 or subsequent CP or in accelerated phase.

3. Clinical diagnosis of HD: induction failure or relapse and sensitive to last
chemotherapy course.

4. Clinical diagnosis of NHL induction failure or relapse and sensitive to last
chemotherapy course.

5. Clinical diagnosis of MDS with intermediate 2- or high-risk IPSS score.

Exclusion Criteria:

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

2. HIV positive.

3. Pregnancy or lactation.

4. Uncontrolled bacterial, fungal or viral infection.

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

6. Availability of appropriate related and willing stem cell donor, who is HLA-matched
at 5 or 6/6 antigens.

7. Prior allogeneic cell transplant.

8. Allergy to bovine or to any product, which may interfere with the treatment.

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

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Overall 100-day mortality

Outcome Time Frame:

100 days

Safety Issue:

Yes

Principal Investigator

Ka Wah Chan, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Texas Transplant Institute

Authority:

United States: Food and Drug Administration

Study ID:

GC P#02.01.001

NCT ID:

NCT00469729

Start Date:

October 2007

Completion Date:

December 2013

Related Keywords:

  • Hematologic Malignancies
  • Acute Myeloid Leukemia
  • Lymphoid Leukemia
  • Chronic Myeloid Leukemia
  • Hodgkin's Disease
  • Non-Hodgkin's Lymphoma
  • Myelodysplastic Syndromes
  • Tetraethylenepentamine
  • Umbilical Cord Blood Stem Cell Transplantation
  • Hematological Malignancies
  • Acute Lymphoid Leukemia
  • Subjects with high-risk hematologic malignancies who are candidates for allogeneic stem cell transplantation
  • Neoplasms
  • Hodgkin Disease
  • Leukemia
  • Leukemia, Lymphoid
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms

Name

Location
Children's Hospital of Orange County Orange, California  92668
Mount Sinai Medical Center New York, New York  10029
Case Western Reserve University Cleveland, Ohio  44106
The Western Pennsylvania Hospital Pittsburgh, Pennsylvania  15224
Texas Transplant Institute San Antonio, Texas  78229
The Cancer Center at Hackensack University Medical Center Hackensack, New Jersey  07601
UCLA's Jonsson Comprehensive Cancer Center Los Angeles, California  90095-1781
The Children's Hospital, B115, University of Colorado Health Sciences Center Aurora, Colorado  80045
Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital Chicago, Illinois  60614
Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine Maywood, Illinois  60153
Steven and Alexandra Cohen Children's Medical Center of New York New York, New York  
Cornell University, Joan & Sanford I. Weill Medical College New York, New York  NY 10065
University of Pittsburgh Cancer Institute/UPMC Cancer Centers Pittsburgh, Pennsylvania  15232
University of Virginia, Hematopoietic Stem Cell Transplant Program West Complex 1300 Jefferson Park Av, Charlottesville, Virginia  22903
Medical College of Wisconsin Pediatric Blood and Marrow Transplant Program Milwaukee, Wisconsin  53226
Medical College of Wisconsin Division of Neoplastic Diseases and Related Disorders Milwaukee, Wisconsin  53226