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Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard Umbilical Cord Blood in Patients With Leukemia, Lymphoma, and Myelodysplastic Syndrome (MDS)

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ClinicalTrials.gov Identifier: NCT02730299
Recruitment Status : Recruiting
First Posted : April 6, 2016
Last Update Posted : August 13, 2019
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd

Brief 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.

Condition or disease Intervention/treatment Phase
Hematological Malignancies Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Chronic Myelogenous Leukemia (CML) Myelodysplastic Syndrome (MDS) Lymphoma Acute Leukemia Drug: NiCord® (omidubicel) Other: Cord Blood Unit Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : November 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020


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




Primary Outcome Measures :
  1. The time to neutrophil engraftment in participants following transplantation. [ Time Frame: must occur on or before 42 days post transplant ]


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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02730299


Contacts
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Contact: Kelly Myers +972-2-6595631 clinicaltrials@gamida-cell.com

  Show 45 Study Locations
Sponsors and Collaborators
Gamida Cell ltd
Investigators
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Study Chair: Mitchell Horwitz, MD Duke University

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Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT02730299     History of Changes
Other Study ID Numbers: GC P#05.01.020
First Posted: April 6, 2016    Key Record Dates
Last Update Posted: August 13, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
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Neoplasms
Neoplasms by Histologic Type
Lymphoma
Syndrome
Leukemia
Myelodysplastic Syndromes
Preleukemia
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Acute
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Myeloproliferative Disorders