Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy - Full Text View

Sponsor:	Gamida Cell -Teva Joint Venture Ltd.
Information provided by (Responsible Party):	Gamida Cell -Teva Joint Venture Ltd.
ClinicalTrials.gov Identifier:	NCT00469729

Purpose

The purpose of this study is to determine the efficacy and safety of transplanting StemEx® in patients with certain hematological malignancies. For these patients, it is suggested that StemEx® can improve upon the outcome of transplanting a single, unmanipulated cord blood unit by significantly increasing the number of stem/progenitor cells available to the patient.

Condition	Intervention	Phase
Hematologic Malignancies Acute Myeloid Leukemia Lymphoid Leukemia Chronic Myeloid Leukemia Hodgkin's Disease Non-Hodgkin's Lymphoma Myelodysplastic Syndromes	Drug: StemEx®	Phase II Phase III

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	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

Resource links provided by NLM:

MedlinePlus related topics: Acute Lymphocytic Leukemia Acute Myeloid Leukemia Cancer Chronic Lymphocytic Leukemia Chronic Myeloid Leukemia Hodgkin Disease Leukemia Lymphoma Myelodysplastic Syndromes

U.S. FDA Resources

Further study details as provided by Gamida Cell -Teva Joint Venture Ltd.:

Primary Outcome Measures:

Overall 100-day mortality [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]
Safety and tolerability measures: The incidence and frequency of adverse experiences, acute toxicity, laboratory data and vital signs follow-up. [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]
Proportion of overall mortality at 1 year [ Time Frame: One year post transplant ] [ Designated as safety issue: Yes ]
Proportion of overall mortality at 2 years [ Time Frame: Two years post transplant ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	100
Study Start Date:	October 2007
Estimated Study Completion Date:	December 2013
Estimated Primary Completion Date:	May 2013 (Final data collection date for primary outcome measure)

Intervention Details:

The stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood cells, which is infused to subject at a rate of 1-3 ml/min in combination with non-manipulated cells derived from the same cord blood unit.

Detailed Description:

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:

Screening phase includes subjects' clinical assessment and screening tests
Conditioning phase includes the myeloablative treatment prior transplantation procedure
Transplantation and post-transplant follow-up phase to day 180
Observational phase: survival status follow-up to day 730 (18 months)

Eligibility

Ages Eligible for Study:	12 Years to 55 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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.
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.
Clinical diagnosis of HD: induction failure or relapse and sensitive to last chemotherapy course.
Clinical diagnosis of NHL induction failure or relapse and sensitive to last chemotherapy course.
Clinical diagnosis of MDS with intermediate 2- or high-risk IPSS score.

Exclusion Criteria:

Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except Hydroxyurea).
HIV positive.
Pregnancy or lactation.
Uncontrolled bacterial, fungal or viral infection.
Subjects with signs and symptoms of active central nervous system (CNS) disease.
Availability of appropriate related and willing stem cell donor, who is HLA-matched at 5 or 6/6 antigens.
Prior allogeneic cell transplant.
Allergy to bovine or to any product, which may interfere with the treatment.
Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00469729

Contacts

Contact: David Snyder, PhD

972-2-6595666

David.snyder@gamida-cell.com

Show 28 Study Locations

Sponsors and Collaborators

Gamida Cell -Teva Joint Venture Ltd.

Investigators

Principal Investigator:	Ka Wah Chan, MD	Texas Transplant Institute
Principal Investigator:	Scott D Rowley, MD	The Cancer Center at Hackensack University Medical Center
Principal Investigator:	Mary Territo, MD	UCLA Oncology Center
Principal Investigator:	Patrick Stiff, MD	Loyola University Cardinal Bernardin Cancer Center
Principal Investigator:	Agha Mounzer, MD	University of Pittsburgh Cancer Institute/UPMC Cancer Centers
Principal Investigator:	Entezam Sahovic, MD	The Western Pennsylvania Hospital
Principal Investigator:	Celia Grosskreutz, MD	Mount Sinai School of Medicine
Principal Investigator:	Roger Giller, MD	The Children's Hospital, B115, University of Colorado Health Sciences Center
Principal Investigator:	Steven Neudorf, MD	Children’s Hospital of Orange County
Principal Investigator:	Ronit Yerushalmi, MD	Chaim Sheba Medical Center
Principal Investigator:	Tsila Zuckerman, MD	Rambam Health Care Campus
Principal Investigator:	Christelle Ferra, MD	Hospital Germans Trias i Pujol
Principal Investigator:	Cristina Arbona, MD	University of Valencia
Principal Investigator:	Guillermo Sanz, MD	Hospital Universitario La Fe
Principal Investigator:	William Arcese, MD	Universita di Roma Tor Vergata
Principal Investigator:	Alberto Bosi, MD	Ospedale di Careggi BMT Unit Department of Haematology
Principal Investigator:	Sonali Chaudhury, MD	Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital
Principal Investigator:	Jorge Sierra, MD	Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Principal Investigator:	Igor B. Resnick, MD, PhD	Department of Bone Marrow Transplantation And Cancer Immunotherapy Hebrew University Hospital Ein-Karem, Jerusalem
Principal Investigator:	Prof. Franco Locatelli, MD	Ospedale Pedriatrico Bambino Gesù
Principal Investigator:	Dr. Mi Kwon, MD	Hospital General Universitario Gregorio Marañón
Principal Investigator:	Dr. Pere Barba, MD	Hospital Universitario Vall d´Hebrón
Principal Investigator:	Dr. Cristina Diaz de Heredia, MD	Hospital Universitario Vall d´Hebrón
Principal Investigator:	Prof. Mary J Laughlin, MD	Hematopoietic Stem Cell Transplant Program, University of Virginia

Responsible Party:	Gamida Cell -Teva Joint Venture Ltd.
ClinicalTrials.gov Identifier:	NCT00469729 History of Changes
Obsolete Identifiers:	NCT00763334
Other Study ID Numbers:	GC P#02.01.001
Study First Received:	May 3, 2007
Last Updated:	January 12, 2012
Health Authority:	United States: Food and Drug Administration