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Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies

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: NCT01221857
Recruitment Status : Completed
First Posted : October 15, 2010
Results First Posted : February 26, 2019
Last Update Posted : April 23, 2019
Information provided by (Responsible Party):
Gamida Cell ltd

Brief Summary:
Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second, Unmanipulated CBU in Patients with Hematological Malignancies

Condition or disease Intervention/treatment Phase
Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Myelodysplastic Syndrome (MDS) Non-Hodgkin's Lymphoma Hodgkin's Disease Drug: NiCord® Phase 1 Phase 2

Detailed Description:

Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative procedure for various hematological malignancies, bone marrow failure syndromes and inherited metabolic disorders. The application of allogeneic HSCT is limited by donor availability such that only approximately one-third of the otherwise appropriate candidates have suitably matched family donors. Alternative donors include mismatched family members or matched unrelated donors, but these approaches are often complicated by an increased risk of graft-versus-host disease (GvHD) and a prolonged and cumbersome search and procurement process. In addition, far fewer subjects of racial minorities find suitable human leukocyte antigen (HLA)-matched donors.

Umbilical cord blood has been increasingly used as an alternative source of stem cells and has extended the availability of allogeneic HSCT to patients who would otherwise not be eligible for this curative approach. In the last decade the number of cord blood transplantations from related and unrelated donors has increased dramatically. It is estimated that more than 20,000 patients have undergone cord blood transplantation from unrelated donors to date for a variety of genetic, hematological, immunological, metabolic and oncologic disorders. The major advantages of cord blood transplantation include easy procurement, no risk to donors, reduced incidence of transmitting infections, immediate availability, and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch. Nevertheless, the low cell dose remains a main limitation of this cell source leading to delayed hematopoietic reconstitution, higher risk of graft failure and relatively high treatment related mortality rates as compared to other hematopoeitic cell sources. To improve outcomes and extend applicability of cord blood transplantation, one potential solution is ex vivo expansion of cord blood-derived stem and progenitor cells.

The Sponsor has undertaken to develop NiCord®, which is based on a novel technology for 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 broader application of umbilical cord blood transplantation and improve clinical outcomes in subjects with high-risk hematological malignancies.

The main objective of the current study is to evaluate the safety of co-transplantation of NiCord® and an unmanipulated cord blood unit in patients with hematological malignancies following myeloablative therapy.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
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 Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies
Study Start Date : November 2010
Actual Primary Completion Date : September 2012
Actual Study Completion Date : May 2013

Arm Intervention/treatment
Experimental: NiCord Drug: NiCord®
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.

Primary Outcome Measures :
  1. Acute Toxicity Associated With the Infusion of NiCord [ Time Frame: 180 days post-transplant ]
    Acute toxicity associated with the infusion of NiCord will be measured by adverse events within 24 hours post-infusion, defined as the acute toxicity period. Known adverse events associated with myeloablation and cord blood transplant were specifically monitored including fever, chills, allergic reaction/hypersensitivity, anaphylaxis, sinus bradycardia, sinus tachycardia, hypertension, hypotension, nausea, vomiting, diarrhea, dyspnea, hypoxia, hemoglobinuria, infection, flank pain and any other skin, CNS, cardiac, pulmonary or other toxicity manifestations.

  2. Proportion of Patients With Neutrophil Engraftment [ Time Frame: 42 days ]
    Neutrophil engraftment was defined as achieving an Absolute Neutrophil Count (ANC) of ≥500 mm3 for 3 consecutive measurements on different days by day 42 inclusive (the day of engraftment was defined as the first of these 3 days). The ANC recovery must be of donor origin documented by peripheral blood chimerism assays indicating less than or equal to 10% host cells in peripheral blood.

Secondary Outcome Measures :
  1. Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV [ Time Frame: 180 days ]

    Acute GvHD was assessed from transplantation (day 0) until day 99 post-transplant or more frequently as clinically indicated. GvHD was classified according to the Glucksberg Classification (Glucksberg, Storb et al. 1974).

    The overall grade of GvHD, however, was determined by an assessment of skin disease, liver disease and gastrointestinal manifestations.

  2. Non-relapse Mortality [ Time Frame: 100 days ]
    Proportion of patients who had non-relapse mortality at 100 days.

Information from the National Library of Medicine

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Ages Eligible for Study:   8 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 related donor able to donate
  • Prior allogeneic HSCT
  • Lymphoma patients with progressive disease
  • Other active malignancy
  • Human immunodeficiency virus (HIV) infection
  • 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): NCT01221857

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United States, Illinois
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States, 60153
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Gamida Cell ltd
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Study Director: David Snyder, PhD Gamida Cell ltd
Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Mitchell Horwitz, MD Duke University
Principal Investigator: Patrick Stiff, MD Loyola University

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Gamida Cell ltd Identifier: NCT01221857     History of Changes
Other Study ID Numbers: GC P#01.01.020
First Posted: October 15, 2010    Key Record Dates
Results First Posted: February 26, 2019
Last Update Posted: April 23, 2019
Last Verified: February 2019

Keywords provided by Gamida Cell ltd:
Double Umbilical Cord Blood Stem Cell Transplantation
Hematological Malignancies
HLA Mismatched Donors
Cord Blood Transplantation

Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Lymphoma, Non-Hodgkin
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Hodgkin Disease
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases