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NCT Number: NCT02730299

Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS

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.

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Key information

Conditions

Hematological Malignancies Acute Leukemia Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Bone Marrow Diseases Chronic Disease Chronic Myelogenous Leukemia (CML) Disease Attributes Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid Leukemia, Myeloid, Acute Lymphatic Diseases Lymphoma Lymphoproliferative Disorders Myelodysplastic Syndrome (MDS) Myelodysplastic Syndromes Myeloproliferative Disorders Neoplasms Neoplasms by Histologic Type Neoplasms by Site Pathologic Processes Pathological Conditions, Signs and Symptoms Precursor Cell Lymphoblastic Leukemia-Lymphoma

Age range

12 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA, Rio de Janeiro, Brazil

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

NiCord® (omidubicel)

Drug

Cord Blood Unit

Other

Cord blood unit

Primary outcomes

  1. Time to Neutrophil Engraftment

    Time frame: post-transplant up to 42 days

    The time to engraftment of neutrophils >500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.

Secondary outcomes

  1. First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation

    Time frame: 100 days post-transplant

    First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population

  2. Days Alive and Out of Hospital in the First 100 Days Post-transplantation

    Time frame: 100 days post-transplantation

    Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population

  3. Number of Participants With Platelet Engraftment by 42 Days Post-transplantation

    Time frame: 42 days post-transplantation

    Platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count > 20 × 10^9/L with no platelet transfusions during the preceding seven days (counting day of engraftment as one of the preceding seven days) for the Intent to Treat Population

Other outcomes

  1. LTF Chimerism

    Time frame: By 5 years post-transplant

    Kaplan Meier probability of donor chimerism <95%

  2. LTF Overall Survival

    Time frame: By 5 years post-transplant

    Kaplan Meier probability of overall survival

  3. LTF Disease Progression/Relapse

    Time frame: By 5 years post-transplant

    Number of patients with relapse during the long-term follow-up period

  4. LTF Chronic GvHD

    Time frame: By 5 years post-transplant

    Cumulative Incidence of Chronic GvHD

Sponsors and collaborators

Lead sponsor

Gamida Cell ltd

Industry

Registry information

Official study 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

Important dates

Study start
2016
Primary completion
2021
Study completion
2025
First posted
Apr 6, 2016
Registry last updated
Feb 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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