Last updated on April 2018

Transplantation of Ex Vivo Expanded UCB-derived Stem & Progenitor Cells vs. Unmanipulated UCB for HM Patients

Brief description of study

This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.

Detailed Study Description

Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.

A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.

The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation.

NiCord is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of Hematopoietic Progenitor Cells (HPC) expanded in ex vivo cultures. The chief aim of the study is to compare the safety and efficacy of NiCord single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.

Clinical Study Identifier: NCT02730299

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Patrick Stiff, MD

Loyola University, Cardinal Bernardin Cancer Center
Maywood, IL United States
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Joseph McGuirk, MD

University of Kansas Cancer Center
Westwood, KS United States
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Corey Cutler, MD

Dana-Farber Cancer Institute
Boston, MA United States
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Claudio Brunstein, MD

University of Minnesota Masonic Cancer Center
Minneapolis, MN United States
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Mitchell Horwitz, MD

Duke University Medical Center
Durham, NC United States
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Rabi Hanna, MD

Cleveland Clinic Children's
Cleveland, OH United States
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Jaap Jan Boelens, MD

University Medical Center Utrecht
Utrecht, Netherlands
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Liang Piu Koh, MD

National University Cancer Institute
Singapore, Singapore
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William Hwang Ying Khee, MD

Singapore General Hospital
Singapore, Singapore
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David Valcárcel, MD, PhD

University Hospital Vall d'Hebron
Barcelona, Spain
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Guillermo Sanz, MD

Hospital Universitario La Fe
Valencia, Spain
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