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

July 27, 2023 updated by: Gamida Cell ltd

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Actual)

125

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
      • Rio De Janeiro, Brazil, 20230-130
        • Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
      • São Paulo, Brazil
        • Hospital Israelita Albert Einstein
      • São Paulo, Brazil, 05403-010
        • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
      • São Paulo, Brazil, 05403-010
        • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
      • São Paulo, Brazil, 14048-900
        • Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto da Universidade de Sao Paulo
  • France
      • Paris, France, 75019
        • Robert Debre
  • Israel
      • Haifa, Israel
        • Rambam
      • Jerusalem, Israel
        • Hadassah Medical Center
      • Petach Tikva, Israel
        • Rabin Medical Center, Beilinson Hospital
      • Tel Aviv, Israel
        • Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
      • Tel Aviv, Israel
        • Tel-Aviv Sourasky Medical Center
      • Tel HaShomer, Israel
        • Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
  • Italy
      • Florence, Italy, 50134
        • Careggi University Hospital
      • Rome, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù
  • Netherlands
      • Utrecht, Netherlands, 3503 AB
        • University Medical Center Utrecht
      • Utrecht, Netherlands, 3584 CS
        • Prinses Maxima Centrum voor Kinderoncologie B.V.
  • Portugal
      • Lisbon, Portugal, 1099-023
        • Instituto Portugues de Oncologia de Lisboa Francisco Gentil
  • Singapore
      • Singapore, Singapore, 169608
        • Singapore General Hospital
      • Singapore, Singapore, 119074
        • National University Cancer Institute
  • Spain
      • Barcelona, Spain
        • Hospital de la Santa Creu i Sant Pau
      • Barcelona, Spain, 08035
        • University Hospital Vall d'Hebron
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron pediatrics
      • Barcelona, Spain, 08908
        • ICO Bellvitge
      • Barcelona, Spain, 08950
        • Sant Joan de Deu
      • Sevilla, Spain
        • Hospital Universitario Virgen del Rocio
      • Valencia, Spain, 46009
        • Hospital Universitario La Fe
      • Valencia, Spain
        • Hospital Universitario y Politécnico La Fe (pediatric)
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
        • Queen Elizabeth Hospital
      • Leeds, United Kingdom, LS9 7TF
        • St James Hospital
      • Manchester, United Kingdom, M13 9WL
        • Manchester University NHS Foundation Trust
    • Surrey
      • Sutton, Surrey, United Kingdom, SM2 5PT
        • The Royal Marsden NHS Foundation Trust
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • UCLA
      • Los Angeles, California, United States, 91010
        • City of Hope
      • Palo Alto, California, United States
        • Stanford University Cancer Institute
      • San Diego, California, United States, 92093
        • UC San Diego Moores Cancer Center
    • Colorado
      • Denver, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Illinois
      • Evanston, Illinois, United States, 60208
        • Northwestern University
      • Maywood, Illinois, United States, 60153
        • Loyola University, Cardinal Bernardin Cancer Center
    • Kansas
      • Westwood, Kansas, United States, 66205
        • University of Kansas Cancer Center
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • The University of Maryland Medicine Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana-Farber Cancer Institute
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Detroit, Michigan, United States
        • Henry Ford Medical Center
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota Masonic Cancer Center
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Rutgers Cancer Institute of New Jersey
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Children's
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh
    • Tennessee
      • Germantown, Tennessee, United States, 38138
        • West Cancer Clinic
    • Texas
      • Dallas, Texas, United States, 75235
        • Children's Medical Center of Dallas
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years to 63 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NiCord® (omidubicel)

NiCord® is a cryopreserved stem/progenitor cell based product comprised of:

  1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF))
  2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells.

Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.
Drug: NiCord® (omidubicel)
Active Comparator: Unmanipulated CBU(s)
Other: Cord Blood Unit
Cord blood unit

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
post-transplant up to 42 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
100 days post-transplant
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
100 days post-transplantation
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
42 days post-transplantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gamida Cell ltd

Investigators

  • Study Chair: Mitchell Horwitz, MD, Duke University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 16, 2016

Primary Completion (Actual)

April 29, 2021

Study Completion (Estimated)

February 18, 2025

Study Registration Dates

First Submitted

March 28, 2016

First Submitted That Met QC Criteria

March 31, 2016

First Posted (Estimated)

April 6, 2016

Study Record Updates

Last Update Posted (Actual)

July 28, 2023

Last Update Submitted That Met QC Criteria

July 27, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GC P#05.01.020

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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