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)
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall 100-day mortality
100 days
Yes
Ka Wah Chan, MD
Principal Investigator
Texas Transplant Institute
United States: Food and Drug Administration
GC P#02.01.001
NCT00469729
October 2007
December 2013
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 |