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

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.

Timeline

Start

2007-10

Primary completion

2013-02

Completion

2015-06

Outcome

Met primary endpoint

paper The 100-day survival was 84.2% for the carlecortemcel-L study group versus 74.6% for the DUCBT group (odds ratio, .50; 95% CI, .26 to .95; P = .035). PMID 29477778 ↗

Publications

• Background 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.
• 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.
• 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.
• 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.
• 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.
• 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.
• 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.

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Indications