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Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy (ExCell)
This study is currently recruiting participants.
Verified by Gamida Cell -Teva Joint Venture Ltd., November 2009
First Received: May 3, 2007   Last Updated: November 15, 2009   History of Changes
Sponsor: Gamida Cell -Teva Joint Venture Ltd.
Information provided by: 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: Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
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: Cancer Hodgkin  Disease Leukemia, Adult Acute Leukemia, Adult Chronic Leukemia, Childhood Lymphoma
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:
    Drug: StemEx®
    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:

  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)
  Eligibility

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

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.
  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

  Hide Study Locations
Locations
United States, California
UCLA's Jonsson Comprehensive Cancer Center Recruiting
8-684 Factor Building, Los Angeles, California, United States, 90095-1781
Contact: Dr. Mary Territo, MD     310-825-7768     MTerrito@mednet.ucla.edu    
Principal Investigator: Dr. Mary Territo, MD            
Children's Hospital of Orange County Recruiting
455 S. Main Street, Orange, California, United States, 92868-3874
Contact: Dr. Steven Neudorf, MD     714-289-4592     SNEUDORF@CHOC.ORG    
Principal Investigator: Dr. Steven Neudorf, MD            
United States, Colorado
The Children's Hospital, B115, University of Colorado Health Sciences Center Not yet recruiting
13123 East 16th Avenue, Aurora, Colorado, United States, 80045
Contact: Prof. Roger Giller, MD     720-777-6892     giller.roger@tchden.org    
Principal Investigator: Prof. Roger Giller, MD            
United States, Illinois
Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine Recruiting
Maywood, Illinois, United States, 60153
Contact: Dr. Patrick Stiff, MD     708-327-3148     pstiff@lumc.edu    
Principal Investigator: Dr. Patrick Stiff, MD            
Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital Recruiting
2300 Children's Plaza, Box #30, Chicago, Illinois, United States, 60614
Contact: Dr. Sonali Chaudhury, MD     773-880-6941     SChaudhury@childrensmemorial.org    
Principal Investigator: Dr. Sonali Chaudhury, MD            
United States, New Jersey
The Cancer Center at Hackensack University Medical Center Recruiting
20 Prospect Ave. Hackensack, New Jersey, United States, 07601
Contact: Dr. Scott D Rowley, MD     201-996-5849     SRowley@humed.com    
Principal Investigator: Dr. Scott D Rowley, MD            
United States, New York
Columbia University College of Physicians and Surgeons Recruiting
3959 Broadway, Chn-1007, New York, New York, United States, 10032
Contact: Prof. Monica Bhatia, MD     +1-212-305-9138     mb2476@columbia.edu    
Principal Investigator: Prof. Monica Bhatia, MD            
Mount Sinai Medical Center Recruiting
One Gustave L Levy Place, Box 1410, New York, New York, United States, 10029
Contact: Prof. Celia Grosskreutz, MD     +1-212-241-6021     Celia.Grosskreutz@msnyuhealth.org    
Principal Investigator: Prof. Celia Grosskreutz, MD            
United States, North Carolina
Duke University Medical Center Recruiting
2400 Pratt Street, Durham, North Carolina, United States, 27705
Contact: Prof. Joanne Kurtzberg, MD     919-668-1119     kurtz001@mc.duke.edu    
Principal Investigator: Prof. Joanne Kurtzberg, MD            
United States, Ohio
Case Western Reserve University Recruiting
10900 Euclid Ave, Wolstein Wrb2-129, Cleveland, Ohio, United States, 10900
Contact: Prof. Mary J Laughlin     (216) 368-5693     mary.laughlin@case.edu    
Principal Investigator: Prof.Mary J Laughlin, MD            
United States, Pennsylvania
University of Pittsburgh Cancer Institute/UPMC Cancer Centers Recruiting
5150 Centre Avenue, Pittsburgh, Pennsylvania, United States, 15232
Contact: Dr. Agha Mounzer, MD     412-235-1052     aghame@upmc.edu    
Principal Investigator: Dr. Agha Mounzer, MD            
The Western Pennsylvania Hospital Recruiting
4800 Friendship Avenue, Pittsburgh, Pennsylvania, United States, 15224
Contact: Dr. Entezam Sahovic, MD     412-578-4484     esahovic@hotmail.com    
Principal Investigator: Dr. Entezam Sahovic, MD            
United States, Texas
Texas Transplant Institute Recruiting
8201 Ewing Halsell, San Antonio, Texas, United States, 78229
Contact: Dr. Ka W Chan, MD     210-575-8500     Kawah.Chan@MHShealth.com    
Principal Investigator: Dr. Ka W Chan, MD            
France
Hopital Saint Louis Not yet recruiting
1, Avenue Claude-Vellefaux, Paris, France, 75010
Contact: Dr. Gerard Socie, MD     +33-1-42-49-96-39     gerard.socie@sls.aphp.fr    
Principal Investigator: Dr. Gerard Socie, MD            
Hungary
Szent Laszlo and Szent Istvan Hospital Recruiting
Budapest,, Hungary, 1097
Contact     +36-1-455-8216     tmasszi@laszlokorhaz.hu    
Principal Investigator: Dr. Tamas Masszi, MD            
Israel
Chaim Sheba Medical Center Recruiting
Tel Hashomer, Israel, 52621
Contact: Dr. Ronit Yerushalmi, MD     972-3-5305340        
Principal Investigator: Dr. Ronit Yerushalmi, MD            
Rambam Medical Center Recruiting
Po Box 9602, Haifa, Israel, 31096
Contact: Dr. Tsila Zuckerman, MD     +972-4-854-2541     t_zuckerman@rambam.health.gov.il    
Principal Investigator: Dr. Tsila Zuckerman, MD            
Hebrew University Hospital Ein-Karem, Department of Bone Marrow Transplantation And Cancer Immunotherapy Recruiting
Jerusalem, Israel, P.O.B 12000
Contact: Prof. Igor B. Resnick, M.D., Ph.D.     (972-2) 677 8354     gashka@hadassah.org.il    
Principal Investigator: Prof. Igor B. Resnick, MD, PhD            
Italy
Universita di Roma Tor Vergata Recruiting
Via Oxford 81, Roma, Italy, 00133
Contact: Prof. William Arcese, MD     +39-333-481-1812     william.arcese@ptvonline.it    
Principal Investigator: Prof. William Arcese, MD            
Ospedale di Careggi BMT Unit Department of Haematology Recruiting
Viale Morgagni, Florence, Italy, 85 - 50134
Contact: Prof. Alberto Bosi, MD     +39-333-410-8756     alberto.bosi@unifi.it    
Principal Investigator: Prof. Alberto Bosi, MD            
Spain
Hospital Germans Trias i Pujol Recruiting
Carretera de Canyet S/n, Badalona, Spain, 08916
Contact: Dr. Christelle Ferra, MD     +34-93-497-89-87     CHRISTELLE.FERRA@telefonica.net    
Principal Investigator: Dr. Christelle Ferra, MD            
Hospital de la Santa Creu i Sant Pau Recruiting
C/ Sant Antoni Maria Claret, Barselona, Spain, 167 - 08025
Contact: Dr. Jorge Sierra, MD     +34-93-556-5647     jsierra@santpau.es    
Principal Investigator: Dr. Jorge Sierra, MD            
Hospital Clinic of Barcelona Recruiting
Villarroel 170, Barcelona, Spain, 08036
Contact: Dr. Enric Carreras, MD     +34-93-227-5428     enric.carreras@fcarreras.es    
Principal Investigator: Dr. Enric Carreras, MD            
Hospital Universitario La Fe Recruiting
Av Campanar 21, Valencia, Spain, 46009
Contact: Dr. Guillermo Sanz, MD     +34-96-386-2709     sanz_gui@gva.es    
Principal Investigator: Dr. Guillermo Sanz, MD            
Spain, Comunidad Valenciana
Hospital Clínico Universitario de Valencia Recruiting
Avda. Blasco Ibañez, 17, Valencia, Comunidad Valenciana, Spain, 46010
Contact: Dr. Cristina Arbona, MD     +34-96-398-7832     arbona_cri@gva.es    
Principal Investigator: Dr. Cristina Arbona, MD            
Sponsors and Collaborators
Gamida Cell -Teva Joint Venture Ltd.
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
  More Information

Additional Information:
Sponsor's website  This link exits the ClinicalTrials.gov site
Study website  This link exits the ClinicalTrials.gov site
Patients Against Lymphoma  This link exits the ClinicalTrials.gov site
Bone and Marrow Transplant Information Network  This link exits the ClinicalTrials.gov site
NCI Leukemia Homepage  This link exits the ClinicalTrials.gov site
Leukemia information on MediciNet.com  This link exits the ClinicalTrials.gov site
American Society for Blood and Marrow Transplantation  This link exits the ClinicalTrials.gov site
American Society of Hematology  This link exits the ClinicalTrials.gov site
Lymphoma Research Foundation  This link exits the ClinicalTrials.gov site
American Cancer Society website  This link exits the ClinicalTrials.gov site
Cord Blood Forum  This link exits the ClinicalTrials.gov site
Center for International Blood and Marrow Transplant Research  This link exits the ClinicalTrials.gov site
National Marrow Donor Program  This link exits the ClinicalTrials.gov site
Children's Hospital of Orange County  This link exits the ClinicalTrials.gov site
University of Colorado Cancer Center  This link exits the ClinicalTrials.gov site
Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital website  This link exits the ClinicalTrials.gov site
Cardinal Bernadin Cancer Center  This link exits the ClinicalTrials.gov site
The Cancer Center at Hackensack University Medical Center  This link exits the ClinicalTrials.gov site
Columbia University College of Physicians and Surgeons  This link exits the ClinicalTrials.gov site
Mount Sinai Medical Center  This link exits the ClinicalTrials.gov site
Duke University Medical Center  This link exits the ClinicalTrials.gov site
Case Western Reserve University  This link exits the ClinicalTrials.gov site
University of Pittsburgh Cancer Institute  This link exits the ClinicalTrials.gov site
The Western Pennsylvania Hospital  This link exits the ClinicalTrials.gov site
MD Anderson Cancer Center  This link exits the ClinicalTrials.gov site
Texas Transplant Institute  This link exits the ClinicalTrials.gov site
Hôpital Saint Louis  This link exits the ClinicalTrials.gov site
Szent Laszlo and Szent Istvan Hospital  This link exits the ClinicalTrials.gov site
Chaim Sheba Medical Center  This link exits the ClinicalTrials.gov site
Rambam Medical Center  This link exits the ClinicalTrials.gov site
Universita di Roma Tor Vergata  This link exits the ClinicalTrials.gov site
Ospedale di Careggi BMT Unit Department of Haematology  This link exits the ClinicalTrials.gov site
Hospital Germans Trias i Pujol  This link exits the ClinicalTrials.gov site
Hospital Clinic of Barcelona  This link exits the ClinicalTrials.gov site
Hospital de la Santa Creu i Sant Pau  This link exits the ClinicalTrials.gov site
Hospital Clínico Universitario de Valencia  This link exits the ClinicalTrials.gov site
Hospital Universitario La Fe  This link exits the ClinicalTrials.gov site
ULCA  This link exits the ClinicalTrials.gov site

Publications:
Peled T, Landau E, Prus E, Treves AJ, Nagler A, Fibach E. Cellular copper content modulates differentiation and self-renewal in cultures of cord blood-derived CD34+ cells. Br J Haematol. 2002 Mar;116(3):655-61. Erratum in: Br J Haematol 2002 May;117(2):485.
Peled T, Landau E, Mandel J, Glukhman E, Goudsmid NR, Nagler A, Fibach E. Linear polyamine copper chelator tetraethylenepentamine augments long-term ex vivo expansion of cord blood-derived CD34+ cells and increases their engraftment potential in NOD/SCID mice. Exp Hematol. 2004 Jun;32(6):547-55.
Prus E, Peled T, Fibach E. The effect of tetraethylenepentamine, a synthetic copper chelating polyamine, on expression of CD34 and CD38 antigens on normal and leukemic hematopoietic cells. Leuk Lymphoma. 2004 Mar;45(3):583-9.
Peled T, Mandel J, Goudsmid RN, Landor C, Hasson N, Harati D, Austin M, Hasson A, Fibach E, Shpall EJ, Nagler A. Pre-clinical development of cord blood-derived progenitor cell graft expanded ex vivo with cytokines and the polyamine copper chelator tetraethylenepentamine. Cytotherapy. 2004;6(4):344-55.
Peled T, Glukhman E, Hasson N, Adi S, Assor H, Yudin D, Landor C, Mandel J, Landau E, Prus E, Nagler A, Fibach E. Chelatable cellular copper modulates differentiation and self-renewal of cord blood-derived hematopoietic progenitor cells. Exp Hematol. 2005 Oct;33(10):1092-100.
de Lima M, McMannis J, Gee A, Komanduri K, Couriel D, Andersson BS, Hosing C, Khouri I, Jones R, Champlin R, Karandish S, Sadeghi T, Peled T, Grynspan F, Daniely Y, Nagler A, Shpall EJ. Transplantation of ex vivo expanded cord blood cells using the copper chelator tetraethylenepentamine: a phase I/II clinical trial. Bone Marrow Transplant. 2008 May;41(9):771-8. Epub 2008 Jan 21.

Responsible Party: Gamida Cell - Teva Joint Venture Ltd. ( Dr. David Snyder )
Study ID Numbers: GC P#02.01.001
Study First Received: May 3, 2007
Last Updated: November 15, 2009
ClinicalTrials.gov Identifier: NCT00469729     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Gamida Cell -Teva Joint Venture Ltd.:
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

Additional relevant MeSH terms:
Leukemia, Lymphoid
Hematologic Neoplasms
Precancerous Conditions
Leukemia, Myeloid, Acute
Leukemia
Preleukemia
Pathologic Processes
Neoplasms by Site
Syndrome
Lymphoma
Hodgkin Disease
Neoplasms by Histologic Type
Immunoproliferative Disorders
 Disease
Immune System Diseases
Hematologic Diseases
Myelodysplastic Syndromes
Myeloproliferative Disorders
Leukemia, Myeloid
Lymphatic Diseases
Neoplasms
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Lymphoma, Non-Hodgkin
Lymphoproliferative Disorders
Bone Marrow Diseases

ClinicalTrials.gov processed this record on November 25, 2009



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