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

NCT00469729 | Completed Phase 2 DMC

• 1 other identifier: GC P#02.01.001
• Study Type: interventional
• Target: 101
• Locations: 5 countries
• Sites: 30

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.

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Overall 100-day mortality

  100 days

Secondary Outcomes (4)

• 180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure

  180 days

• Safety and tolerability measures: The incidence and frequency of adverse experiences, acute toxicity, laboratory data and vital signs follow-up.

  180 days

• Proportion of overall mortality at 1 year

  One year post transplant

• Proportion of overall mortality at 2 years

  Two years post transplant

Study Arms (1)

StemEx

EXPERIMENTAL

Drug: StemEx®

Interventions

StemEx® DRUG

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.

StemEx

Eligibility Criteria

• Age: 12 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Gamida Cell -Teva Joint Venture Ltd.: lead

Study Sites (30)

UCLA's Jonsson Comprehensive Cancer Center

Los Angeles, California, 90095-1781, United States

Location

Children's Hospital of Orange County

Orange, California, 92868-3874, United States

Location

The Children's Hospital, B115, University of Colorado Health Sciences Center

Aurora, Colorado, 80045, United States

Location

Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine

Maywood, Illinois, 60153, United States

Location

The Cancer Center at Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Cornell University, Joan & Sanford I. Weill Medical College

New York, New York, NY 10065, United States

Location

Steven and Alexandra Cohen Children's Medical Center of New York

New York, New York, United States

Location

Mount Sinai Medical Center

One Gustave L Levy Place, BOX 1410, New York, New York, 10029, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106, United States

Location

The Western Pennsylvania Hospital

Pittsburgh, Pennsylvania, 15224, United States

Location

University of Pittsburgh Cancer Institute/UPMC Cancer Centers

Pittsburgh, Pennsylvania, 15232, United States

Location

Texas Transplant Institute

San Antonio, Texas, 78229, United States

Location

University of Virginia, Hematopoietic Stem Cell Transplant Program

West Complex 1300 Jefferson Park Av, Charlottesville, Virginia, 22903, United States

Location

Medical College of Wisconsin Division of Neoplastic Diseases and Related Disorders

Milwaukee, Wisconsin, 53226, United States

Location

Medical College of Wisconsin Pediatric Blood and Marrow Transplant Program

Milwaukee, Wisconsin, 53226, United States

Location

Szent Laszlo & Szent Istvan Hospital

Budapest, 1097, Hungary

Location

Hebrew University Hospital Ein-Karem, Department of Bone Marrow Transplantation And Cancer Immunotherapy

Jerusalem, P.O.B 12000, Israel

Location

Rambam Medical Center

PO Box 9602, Haifa, 31096, Israel

Location

Chaim Sheba Medical Center

Tel Litwinsky, 52621, Israel

Location

Ospedale Pedriatrico Bambino Gesù

Roma, 00165, Italy

Location

Universita di Roma Tor Vergata

Via Oxford 81, Roma, 00133, Italy

Location

Ospedale di Careggi BMT Unit Department of Haematology

Viale Morgagni, Florence, 85 - 50134, Italy

Location

Hospital Clínico Universitario de Valencia

Avda. Blasco Ibañez, 17, Valencia, Valencia, 46010, Spain

Location

Hospital Universitario La Fe

Av Campanar 21, Valencia, 46009, Spain

Location

Hospital de la Santa Creu i Sant Pau

C/ Sant Antoni Maria Claret, Barselona, 167 - 08025, Spain

Location

Hospital Germans Trias i Pujol

Carretera de Canyet S/n, Badalona, 08916, Spain

Location

Hospital General Universitario Gregorio Marañón

Doctor Esquerdo 46 , Madrid, 28007, Spain

Location

Hospital Universitario Vall d´Hebrón (Pediatrics)

Passeig de La Vall d´Hebrón 119-129, Barcelona, 08035, Spain

Location

Hospital Universitario Vall d´Hebrón

Passeig de La Vall d´Hebrón 119-129, Barcelona, 08035, Spain

Location

Related Publications (8)

• 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. doi: 10.1046/j.0007-1048.2001.03316.x.

  PMID: 11849228 BACKGROUND

• 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. doi: 10.1016/j.exphem.2004.03.002.

  PMID: 15183895 BACKGROUND

• 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. doi: 10.1080/10428190310001598035.

  PMID: 15160922 BACKGROUND

• 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. doi: 10.1080/14653240410004916.

  PMID: 16146887 BACKGROUND

• 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. doi: 10.1016/j.exphem.2005.06.015.

  PMID: 16219531 BACKGROUND

• 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.

  BACKGROUND

• 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.

  PMID: 18209724 BACKGROUND

• Stiff PJ, Montesinos P, Peled T, Landau E, Goudsmid NR, Mandel J, Hasson N, Olesinski E, Glukhman E, Snyder DA, Cohen EG, Kidron OS, Bracha D, Harati D, Ben-Abu K, Freind E, Freedman LS, Cohen YC, Olmer L, Barishev R, Rocha V, Gluckman E, Horowitz MM, Eapen M, Nagler A, Sanz G. Cohort-Controlled Comparison of Umbilical Cord Blood Transplantation Using Carlecortemcel-L, a Single Progenitor-Enriched Cord Blood, to Double Cord Blood Unit Transplantation. Biol Blood Marrow Transplant. 2018 Jul;24(7):1463-1470. doi: 10.1016/j.bbmt.2018.02.012. Epub 2018 Mar 1.

  PMID: 29477778 DERIVED

Related Links

• Sponsor's website
• Patients Against Lymphoma
• Bone and Marrow Transplant Information Network
• NCI Leukemia Homepage
• Leukemia information on MediciNet.com
• American Society for Blood and Marrow Transplantation
• American Society of Hematology
• Lymphoma Research Foundation
• American Cancer Society website
• Center for International Blood and Marrow Transplant Research
• National Marrow Donor Program
• Children's Hospital of Orange County
• Northwestern University School of Medicine, Stem Cell Transplant Program, Children's Memorial Hospital website
• Duke University Medical Center
• Case Western Reserve University
• University of Pittsburgh Cancer Institute
• MD Anderson Cancer Center
• Texas Transplant Institute
• Hôpital Saint Louis
• Chaim Sheba Medical Center
• Rambam Medical Center
• Universita di Roma Tor Vergata
• Ospedale di Careggi BMT Unit Department of Haematology
• Hospital Germans Trias i Pujol
• Hospital Clinic of Barcelona
• Hospital de la Santa Creu i Sant Pau
• ULCA

MeSH Terms

Conditions

Hematologic Neoplasms; Leukemia, Myeloid, Acute; Leukemia, Lymphoid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Hodgkin Disease; Lymphoma, Non-Hodgkin; Myelodysplastic Syndromes; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Lymphoma

Study Officials

• 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

  Icahn School of Medicine at Mount Sinai

  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

  Germans Trias i Pujol Hospital

  PRINCIPAL INVESTIGATOR

• Cristina Arbona, MD

  Hospital Clínico Universitario de 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2007
• First Posted: May 4, 2007
• Study Start: October 1, 2007
• Primary Completion: February 1, 2013
• Study Completion: June 1, 2015
• Last Updated: July 10, 2015

Record last verified: 2013-03

Locations

HU (1); IT (3); IL (3); US (16); ES (7)