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Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01816230
Recruitment Status : Completed
First Posted : March 22, 2013
Last Update Posted : January 3, 2019
Information provided by (Responsible Party):
Gamida Cell ltd

Brief Summary:
A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies.

Condition or disease Intervention/treatment Phase
Hematological Malignancies Acute Lymphoblastic Leukemia (ALL) Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS) Drug: NiCord® Phase 1 Phase 2

Detailed Description:

Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.

The study is designed as a multi center, single arm study, evaluating the safety and efficacy of the transplantation of NiCord® to patients with hematological malignancies following myeloablative therapy.

Total study duration is approximately 400 days from the signing of informed consent to the last visit one year following transplantation

The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy as follows:

The main study objectives are to assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation and to assess the incidence of secondary graft failure at 180 days following transplantation of NiCord® Ten evaluable patients recruited for the study should be 12-65 years of age, up to a maximum of 15 treated patients.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 43 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescent and Adult Patients With Hematological Malignancies
Study Start Date : April 2013
Actual Primary Completion Date : June 2017
Actual Study Completion Date : June 2018

Arm Intervention/treatment
Experimental: NiCord®
NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells.
Drug: NiCord®

Primary Outcome Measures :
  1. Engraftment [ Time Frame: 42 days ]
    Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.

Information from the National Library of Medicine

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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

Inclusion Criteria:

  • Applicable disease and eligible for myeloablative SCT
  • Patients must have two partially HLA-matched CBUs
  • Back-up stem cell source
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • 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 identifier (NCT number): NCT01816230

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United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
United States, Illinois
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States, 60153
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, New York
Steven and Alexandra Cohen Children's Medical Center
New York, New York, United States, 11040
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Tennessee
Nashville, Tennessee, United States, 37240
Children Hospital Gaslini Scientific Institute
Genoa, Italy, 16147
University of Turin
Turin, Italy, 10043
University Medical Center Utrecht
Utrecht, Netherlands, 3503 AB
National University Cancer Institute
Singapore, Singapore, 119074
Singapore General Hospital
Singapore, Singapore, 169608
University Hospital Vall d´Hebron
Barcelona, Spain, 08035
Hospital Universitario La Fe
Valencia, Spain, 46009
Sponsors and Collaborators
Gamida Cell ltd
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Principal Investigator: Mitchell Horwitz, MD Duke University
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Gamida Cell ltd Identifier: NCT01816230    
Other Study ID Numbers: GC P#03.01.020
First Posted: March 22, 2013    Key Record Dates
Last Update Posted: January 3, 2019
Last Verified: January 2019
Additional relevant MeSH terms:
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Leukemia, Myeloid, Acute
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Hematologic Neoplasms
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Leukemia, Myeloid
Bone Marrow Diseases
Hematologic Diseases
Leukemia, Lymphoid
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms by Site