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Hematologic MalignanciesPhase 2/3ExCellNCT00469729

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

A Phase 2/3 study testing StemEx® in people with Hematologic Malignancies and Acute Myeloid Leukemia, run by Gamida Cell -Teva Joint Venture Ltd. at 30 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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… Read more about this study

Who is it for?
Ages 12 to 55 years
What is being tested?
StemEx®
Could I receive a placebo?
No, everyone receives an active treatment
How many people
101 participants
Where
30 sitesin 5 countries
Study dates
Oct 2007to Jun 2015
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 55 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 9Show fewer
  • 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.

Trial locations and contacts

30 sites in 5 countries.

30 sites in 5 countries

United States16 sites

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

    Aurora, Colorado, 80045

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

    Chicago, Illinois, 60614

  • Case Western Reserve University

    Cleveland, Ohio, 44106

  • The Cancer Center at Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • UCLA's Jonsson Comprehensive Cancer Center

    Los Angeles, California, 90095-1781

  • Cardinal Bernardin Cancer Center, Loyola University Stritch School of Medicine

    Maywood, Illinois, 60153

  • Medical College of Wisconsin Division of Neoplastic Diseases and Related Disorders

    Milwaukee, Wisconsin, 53226

  • Medical College of Wisconsin Pediatric Blood and Marrow Transplant Program

    Milwaukee, Wisconsin, 53226

  • Cornell University, Joan & Sanford I. Weill Medical College

    New York, New York, NY 10065

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

    New York, New York

  • Mount Sinai Medical Center

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

  • Children's Hospital of Orange County

    Orange, California, 92868-3874

  • The Western Pennsylvania Hospital

    Pittsburgh, Pennsylvania, 15224

  • University of Pittsburgh Cancer Institute/UPMC Cancer Centers

    Pittsburgh, Pennsylvania, 15232

  • Texas Transplant Institute

    San Antonio, Texas, 78229

  • University of Virginia, Hematopoietic Stem Cell Transplant Program

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

Other trials in United States

Spain7 sites

  • Hospital Universitario La Fe

    Av Campanar 21, Valencia, 46009

  • Hospital Clínico Universitario de Valencia

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

  • Hospital de la Santa Creu i Sant Pau

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

  • Hospital Germans Trias i Pujol

    Carretera de Canyet S/n, Badalona, 08916

  • Hospital General Universitario Gregorio Marañón

    Doctor Esquerdo 46 , Madrid, 28007

  • Hospital Universitario Vall d´Hebrón (Pediatrics)

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

  • Hospital Universitario Vall d´Hebrón

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

Other trials in Spain

Israel3 sites

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

    Jerusalem, P.O.B 12000

  • Rambam Medical Center

    PO Box 9602, Haifa, 31096

  • Chaim Sheba Medical Center

    Tel HaShomer, 52621

Other trials in Israel

Italy3 sites

  • Ospedale Pedriatrico Bambino Gesù

    Roma, 00165

  • Universita di Roma Tor Vergata

    Via Oxford 81, Roma, 00133

  • Ospedale di Careggi BMT Unit Department of Haematology

    Viale Morgagni, Florence, 85 - 50134

Other trials in Italy

Hungary1 site

  • Szent Laszlo & Szent Istvan Hospital

    Budapest, 1097

Other trials in Hungary

About this 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

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.

Read the 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)
Being tested
StemEx®
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
Medical subject headings
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

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

Study groups in NCT00469729
Study groupRoleWhat they receive
StemExExperimentalNot described
Receives: Drug: StemEx®

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall 100-day mortality

    Measured: 100 days

Secondary outcomes (4)

  1. 180 day mortality, acute Graft versus Host Disease (GvHD) grades III-IV, engraftment failure

    Measured: 180 days

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

    Measured: 180 days

  3. Proportion of overall mortality at 1 year

    Measured: One year post transplant

  4. Proportion of overall mortality at 2 years

    Measured: Two years post transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 May 2007 (estimated)
  2. Study startOct 2007
  3. Primary completionFeb 2013
  4. Study completionJun 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
101 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00469729
Sponsor's protocol ID
GC P#02.01.001
Acronym
ExCell
Status last verified
Mar 2013
First submitted
3 May 2007
Last updated
10 Jul 2015
Results posted
No
Data monitoring committee
Yes

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

    PubMed 11849228

  • 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.

    PubMed 15183895

  • 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.

    PubMed 15160922

  • 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.

    PubMed 16146887

  • 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.

    PubMed 16219531

  • 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.

  • 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.

    PubMed 18209724

  • 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.

    PubMed 29477778

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing StemEx® in people with Hematologic Malignancies and Acute Myeloid Leukemia, run by Gamida Cell -Teva Joint Venture Ltd. at 30 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 30 sites in United States, Spain, Israel, Italy, Hungary. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall 100-day mortality (time frame: 100 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00469729, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2015.

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