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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd
ClinicalTrials.gov Identifier:
NCT01816230
First received: March 19, 2013
Last updated: August 6, 2017
Last verified: August 2017
  Purpose
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 Intervention Phase
Hematological Malignancies Acute Lymphoblastic Leukemia (ALL) Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS) Drug: NiCord® Phase 1 Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Gamida Cell ltd:

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


Estimated Enrollment: 40
Study Start Date: April 2013
Estimated Study Completion Date: June 2018
Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NiCord®
NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells.
Drug: NiCord®

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.

  Eligibility

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

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01816230

Locations
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
Vanderbilt
Nashville, Tennessee, United States, 37240
Italy
Children Hospital Gaslini Scientific Institute
Genoa, Italy, 16147
University of Turin
Turin, Italy, 10043
Netherlands
University Medical Center Utrecht
Utrecht, Netherlands, 3503 AB
Singapore
National University Cancer Institute
Singapore, Singapore, 119074
Singapore General Hospital
Singapore, Singapore, 169608
Spain
University Hospital Vall d´Hebron
Barcelona, Spain, 08035
Hospital Universitario La Fe
Valencia, Spain, 46009
Sponsors and Collaborators
Gamida Cell ltd
Investigators
Principal Investigator: Mitchell Horwitz, MD Duke University
  More Information

Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT01816230     History of Changes
Other Study ID Numbers: GC P#03.01.020
Study First Received: March 19, 2013
Last Updated: August 6, 2017

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

ClinicalTrials.gov processed this record on August 18, 2017