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NCT00469729 Clinical Trial

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

Acute Myeloid Leukemia Clinical Trial

— ExCell  

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT00469729
Other study ID # GC P#02.01.001
Secondary ID
Status Completed
Phase Phase 2/Phase 3
First received May 3, 2007
Last updated July 9, 2015
Start date October 2007
Est. completion date June 2015

Study information
Verified date March 2013
Source Gamida Cell -Teva Joint Venture Ltd.
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.

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)

Other known NCT identifiers
  • NCT00763334

Recruitment information / eligibility
Status Completed
Enrollment 101
Est. completion date June 2015
Est. primary completion date February 2013
Accepts healthy volunteers No
Gender Both
Age group 12 Years to 55 Years
Eligibility 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.

Study Design

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

Related Conditions & MeSH terms

Intervention

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.

Locations
Country Name City State
Hungary Szent Laszlo & Szent Istvan Hospital Budapest
Israel Hebrew University Hospital Ein-Karem, Department of Bone Marrow Transplantation And Cancer Immunotherapy Jerusalem
Israel Rambam Medical Center PO Box 9602, Haifa
Israel Chaim Sheba Medical Center Tel Hashomer
Italy Ospedale Pedriatrico Bambino Gesù Roma
Italy Universita di Roma Tor Vergata via Oxford 81, Roma
Italy Ospedale di Careggi BMT Unit Department of Haematology Viale Morgagni, Florence
Spain Hospital Universitario La Fe Av Campanar 21, Valencia
Spain Hospital Clínico Universitario de Valencia Avda. Blasco Ibañez, 17, Valencia Comunidad Valenciana
Spain Hospital de la Santa Creu i Sant Pau C/ Sant Antoni Maria Claret, Barselona
Spain Hospital Germans Trias i Pujol Carretera de Canyet s/n, Badalona
Spain Hospital General Universitario Gregorio Marañón Doctor Esquerdo 46 , Madrid
Spain Hospital Universitario Vall d´Hebrón Passeig de la Vall d´Hebrón 119-129, Barcelona
Spain Hospital Universitario Vall d´Hebrón (Pediatrics) Passeig de la Vall d´Hebrón 119-129, Barcelona
United States The Children's Hospital, B115, University of Colorado Health Sciences Center Aurora Colorado
United States Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital Chicago Illinois
United States Case Western Reserve University Cleveland Ohio
United States The Cancer Center at Hackensack University Medical Center Hackensack New Jersey
United States UCLA's Jonsson Comprehensive Cancer Center Los Angeles California
United States Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine Maywood Illinois
United States Medical College of Wisconsin Division of Neoplastic Diseases and Related Disorders Milwaukee Wisconsin
United States Medical College of Wisconsin Pediatric Blood and Marrow Transplant Program Milwaukee Wisconsin
United States Cornell University, Joan & Sanford I. Weill Medical College New York New York
United States Steven and Alexandra Cohen Children's Medical Center of New York New York New York
United States Mount Sinai Medical Center One Gustave L Levy Place, BOX 1410, New York New York
United States Children's Hospital of Orange County Orange California
United States The Western Pennsylvania Hospital Pittsburgh Pennsylvania
United States University of Pittsburgh Cancer Institute/UPMC Cancer Centers Pittsburgh Pennsylvania
United States Texas Transplant Institute San Antonio Texas
United States University of Virginia, Hematopoietic Stem Cell Transplant Program West Complex 1300 Jefferson Park Av, Charlottesville Virginia

Sponsors (1)
Lead Sponsor Collaborator
Gamida Cell -Teva Joint Venture Ltd.

Countries where clinical trial is conducted

United States,  Hungary,  Israel,  Italy,  Spain, 

References & Publications (7)

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. doi: 10.1038/sj.bmt.1705979. Epub 2008 Jan 21. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Shpall EJ, M.d.L., K. Chan, R. Champlin, A. Gee, P. Thall, K. Komanduri, D. Couriel, C. Hosing, B. Andersson, R. Jones, S. Giralt, S. Karandish, T. Sadeghi, B. Muriera, S. O'Connor, L. Wooten, X. Wang, S. Robinson, P. Fu, J. Wilson, T. Peled, F. Grynspan, A. Nagler, J. McMannis; A Phase I/II Study of Ex Vivo Expanded Cord Blood for Leukemia and Lymphoma. ISCT 2005 - conference publication, 2005.

Outcome
Type Measure Description Time frame Safety issue
Primary Overall 100-day mortality 100 days Yes
Secondary 180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure 180 days Yes
Secondary Safety and tolerability measures: The incidence and frequency of adverse experiences, acute toxicity, laboratory data and vital signs follow-up. 180 days Yes
Secondary Proportion of overall mortality at 1 year One year post transplant Yes
Secondary Proportion of overall mortality at 2 years Two years post transplant Yes
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