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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

Tracking Information
First Submitted Date  ICMJE October 14, 2010
First Posted Date  ICMJE October 15, 2010
Results First Submitted Date  ICMJE May 24, 2017
Results First Posted Date  ICMJE February 26, 2019
Last Update Posted Date April 23, 2019
Study Start Date  ICMJE November 2010
Actual Primary Completion Date September 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 3, 2019)
  • 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.
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: October 14, 2010)
  • Assessment of the acute toxicity associated with the infusion of NiCord [ Time Frame: 180 days post-transplant ]
  • Assessment of the proportion of patients with neutrophil engraftment following co-transplantation of NiCord and unmanipulated cord blood grafts [ Time Frame: 180 days post-transplant ]
Change History Complete list of historical versions of study NCT01221857 on Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 11, 2019)
  • 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.
  • Non-relapse Mortality [ Time Frame: 100 days ]
    Proportion of patients who had non-relapse mortality at 100 days.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 14, 2010)
  • Proportion of non-relapse mortality at 100 days [ Time Frame: 100 days post-transplant ]
  • Proportion of patients who developed acute GvHD grade II-IV and III-IV [ Time Frame: 180 days post-transplant ]
  • Time from infusion of both units to neutrophil and platelet engraftment [ Time Frame: 180 days post-transplant ]
  • Contribution of both NiCord and the unmanipulated CBU to initial and sustained engraftment [ Time Frame: 180 days post-transplant ]
  • Proportion of patients with full donor chimerism (>90%) by day 100 [ Time Frame: 180 days post-transplant ]
  • Immune reconstitution at 60, 100 and 180 days [ Time Frame: 60, 100 and 180 days post-transplant ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies
Official Title  ICMJE 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
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
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myelogenous Leukemia (AML)
  • Myelodysplastic Syndrome (MDS)
  • Non-Hodgkin's Lymphoma
  • Hodgkin's Disease
Intervention  ICMJE Drug: NiCord®
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Study Arms  ICMJE Experimental: NiCord
Intervention: Drug: NiCord®
Publications *

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 14, 2010)
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 2013
Actual Primary Completion Date September 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01221857
Other Study ID Numbers  ICMJE GC P#01.01.020
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Gamida Cell ltd
Study Sponsor  ICMJE Gamida Cell ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE
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
PRS Account Gamida Cell ltd
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP