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

This Tabular View shows the required WHO registration data elements as marked by ^†

Tracking Information

First Received Date ^†

May 3, 2007

Last Updated Date

May 10, 2009

Start Date ^†

October 2007

Current Primary Outcome Measures ^†

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

Original Primary Outcome Measures ^†

Same as current

Change History

Complete list of historical versions of study NCT00469729 on ClinicalTrials.gov Archive Site

Current 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 ]

Original Secondary Outcome Measures ^†

Same as current

Descriptive Information

Brief Title ^†

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

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

Brief Summary

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.

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.

Study Phase

Phase II, Phase III

Study Type ^†

Interventional

Study Design ^†

Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study

Condition ^†

Hematologic Malignancies
Acute Myeloid Leukemia
Lymphoid Leukemia
Chronic Myeloid Leukemia
Hodgkin's Disease
Non-Hodgkin's Lymphoma
Myelodysplastic Syndromes

Intervention ^†

Drug: StemEx®

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^†

Recruiting

Estimated Enrollment ^†

100

Estimated Completion Date

May 2012

Estimated Primary Completion Date

November 2011 (final data collection date for primary outcome measure)

Eligibility 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.

Gender

Both

Ages

12 Years to 55 Years

Accepts Healthy Volunteers

Contacts ^††

Contact: David Snyder, PhD

972-2-6595666

David.snyder@gamida-cell.com

Location Countries ^†

United States, France, Hungary, Israel, Italy, Spain

Expanded Access Status

Administrative Information

NCT ID ^†

NCT00469729

Responsible Party

Dr. David Snyder, Gamida Cell - Teva Joint Venture Ltd.

Secondary IDs ^††

Study Sponsor ^†

Gamida Cell -Teva Joint Venture Ltd.

Collaborators ^††

Investigators ^†

Principal Investigator:	Ka Wah Chan, MD	Texas Transplant Institute
Principal Investigator:	Mary J Laughlin, MD	Case Western Reserve University
Principal Investigator:	Scott D Rowley, MD	The Cancer Center at Hackensack University Medical Center
Principal Investigator:	Mary Territo, MD	UCLA Oncology Center
Principal Investigator:	Joanne Kurtzberg, MD	Duke University
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:	Monica Bhatia, MD	Columbia University College of Physicians and Surgeons
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:	Enric Carreras, MD	Hospital Clinic of Barcelona
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:	Gerard Socie, MD	Hopital Saint Louis
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:	Tamas Masszi, MD	Szent Laszlo and Szent Istvan Hospital
Principal Investigator:	Igor B. Resnick, MD, PhD	Department of Bone Marrow Transplantation And Cancer Immunotherapy Hebrew University Hospital Ein-Karem, Jerusalem

Information Provided By

Gamida Cell -Teva Joint Venture Ltd.

Verification Date

May 2009

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.