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

Sponsor
Gamida Cell ltd (Industry)
Overall Status
Completed
CT.gov ID
NCT01816230
Collaborator
(none)
36
15
1
62
2.4
0

Study Details

Study Description

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.

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
36 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 :
Apr 1, 2013
Actual Primary Completion Date :
Jun 1, 2017
Actual Study Completion Date :
Jun 1, 2018

Arms and Interventions

Arm Intervention/Treatment
Experimental: NiCord®

NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells.

Drug: NiCord®

Outcome Measures

Primary Outcome Measures

  1. Engraftment [42 days]

    Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years to 65 Years
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Cedars-Sinai Medical Center Los Angeles California United States 90048
2 Loyola University, Cardinal Bernardin Cancer Center Maywood Illinois United States 60153
3 University of Minnesota Minneapolis Minnesota United States 55455
4 Steven and Alexandra Cohen Children's Medical Center New York New York United States 11040
5 Duke University Medical Center Durham North Carolina United States 27710
6 Cleveland Clinic Cleveland Ohio United States 44195
7 Oregon Health & Science University Portland Oregon United States 97239
8 Vanderbilt Nashville Tennessee United States 37240
9 Children Hospital Gaslini Scientific Institute Genoa Italy 16147
10 University of Turin Turin Italy 10043
11 University Medical Center Utrecht Utrecht Netherlands 3503 AB
12 National University Cancer Institute Singapore Singapore 119074
13 Singapore General Hospital Singapore Singapore 169608
14 University Hospital Vall d´Hebron Barcelona Spain 08035
15 Hospital Universitario La Fe Valencia Spain 46009

Sponsors and Collaborators

  • Gamida Cell ltd

Investigators

  • Principal Investigator: Mitchell Horwitz, MD, Duke University

Study Documents (Full-Text)

More Information

Publications

None provided.
Responsible Party:
Gamida Cell ltd
ClinicalTrials.gov Identifier:
NCT01816230
Other Study ID Numbers:
  • GC P#03.01.020
First Posted:
Mar 22, 2013
Last Update Posted:
Oct 22, 2020
Last Verified:
Oct 1, 2020

Study Results

Participant Flow

Recruitment Details
Pre-assignment Detail
Arm/Group Title NiCord®
Arm/Group Description NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
Period Title: Overall Study
STARTED 36
COMPLETED 36
NOT COMPLETED 0

Baseline Characteristics

Arm/Group Title NiCord®
Arm/Group Description NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood (UCB) cells. NiCord®
Overall Participants 36
Age (Years) [Median (Full Range) ]
Median (Full Range) [Years]
44
Sex: Female, Male (Count of Participants)
Female
16
44.4%
Male
20
55.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native
0
0%
Asian
2
5.6%
Native Hawaiian or Other Pacific Islander
0
0%
Black or African American
4
11.1%
White
30
83.3%
More than one race
0
0%
Unknown or Not Reported
0
0%
Primary diagnosis (Count of Participants)
Acute lymphoblastic leukemia (ALL)
9
25%
Adult acute myeloid leukemia (AML)
17
47.2%
Myelodysplastic syndromes (MDS)
7
19.4%
non-Hodgkins Lymphoma
0
0%
Hodgkins Disease
1
2.8%
Chronic myeloid leukemia (CML)
2
5.6%
Adjusted disease risk index (Count of Participants)
Low
8
22.2%
Intermediate
15
41.7%
High
13
36.1%

Outcome Measures

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

Outcome Measure Data

Analysis Population Description
[Not Specified]
Arm/Group Title NiCord®
Arm/Group Description NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
Measure Participants 36
Number [percentage of patients]
94

Adverse Events

Time Frame one year
Adverse Event Reporting Description
Arm/Group Title NiCord®
Arm/Group Description NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
All Cause Mortality
NiCord®
Affected / at Risk (%) # Events
Total 16/36 (44.4%)
Serious Adverse Events
NiCord®
Affected / at Risk (%) # Events
Total 34/36 (94.4%)
Immune system disorders
GvHD 11/36 (30.6%)
Infections and infestations
Infection 17/36 (47.2%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hematologic Malignancy 8/36 (22.2%)
Other (Not Including Serious) Adverse Events
NiCord®
Affected / at Risk (%) # Events
Total 36/36 (100%)
Immune system disorders
GvHD 6/36 (16.7%)
Metabolism and nutrition disorders
Decreased appetite 9/36 (25%)
Hyperglycemia 6/36 (16.7%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukemia recurrent 5/36 (13.9%)
Vascular disorders
Hypertension 13/36 (36.1%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Name/Title Kelly Myers
Organization Gamida Cell
Phone 026595631
Email kelly@gamida-cell.com
Responsible Party:
Gamida Cell ltd
ClinicalTrials.gov Identifier:
NCT01816230
Other Study ID Numbers:
  • GC P#03.01.020
First Posted:
Mar 22, 2013
Last Update Posted:
Oct 22, 2020
Last Verified:
Oct 1, 2020