Summary

for people ages 12-65 (full criteria)
at UCLA UCSD
study started
estimated completion
Gary Schiller(ucla)Carolyn Mulroney, MD(ucsd)

Description

Summary

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.

Official Title

A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, UCB-derived, Stem and Progenitor Cells, vs. Unmanipulated UCB for Patients With Hematological Malignancies

Details

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.

Keywords

Hematological Malignancies Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Chronic Myelogenous Leukemia (CML) Myelodysplastic Syndrome (MDS) Lymphoma Acute Leukemia Leukemia Preleukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Acute Myelodysplastic Syndromes Syndrome Neoplasms NiCord® (omidubicel) Cord Blood Unit Unmanipulated CBU(s)

Eligibility

You can join if…

Open to people ages 12-65

  • Applicable disease criteria
  • Patients must have one or two partially HLA-matched CBUs
  • Back-up stem cell source
  • Adequate Karnofsky/Lansky Performance score
  • Sufficient physiological reserves
  • Signed written informed consent

You CAN'T join if...

  • HLA-matched donor able to donate
  • Prior allogeneic HSCT
  • Other active malignancy
  • Active or uncontrolled infection
  • Active/symptoms of central nervous system (CNS) disease
  • Pregnancy or lactation

Locations

  • UCLA accepting new patients
    Los Angeles California 90095 United States
  • UC San Diego Moores Cancer Center accepting new patients
    San Diego California 92093 United States

Lead Scientists

  • Gary Schiller (ucla)
  • Carolyn Mulroney, MD (ucsd)
    Co-Investigator on several clinical trials in the Blood and Marrow Transplantation Division: BMTCTN 0402, IRB # 101006, A Phase III Randomized, Multicenter Trial comparing Sirolimus/Tacrolimus with Tacrolimus/Methotrexate as a Graft-Versus-Host Disease (GVHD) Prophylaxis After HLA- Matched, Related Peripheral Blood Stem Cell transplantation. B.A.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Gamida Cell ltd
ID
NCT02730299
Phase
Phase 3
Study Type
Interventional
Last Updated