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
Enrollment
15
Locations
1
Arm
62
Duration (Months)
2.4
Patients Per Site
0
Patients Per Site Per Month

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

ArmIntervention/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

SiteCityStateCountryPostal Code
1Cedars-Sinai Medical CenterLos AngelesCaliforniaUnited States90048
2Loyola University, Cardinal Bernardin Cancer CenterMaywoodIllinoisUnited States60153
3University of MinnesotaMinneapolisMinnesotaUnited States55455
4Steven and Alexandra Cohen Children's Medical CenterNew YorkNew YorkUnited States11040
5Duke University Medical CenterDurhamNorth CarolinaUnited States27710
6Cleveland ClinicClevelandOhioUnited States44195
7Oregon Health & Science UniversityPortlandOregonUnited States97239
8VanderbiltNashvilleTennesseeUnited States37240
9Children Hospital Gaslini Scientific InstituteGenoaItaly16147
10University of TurinTurinItaly10043
11University Medical Center UtrechtUtrechtNetherlands3503 AB
12National University Cancer InstituteSingaporeSingapore119074
13Singapore General HospitalSingaporeSingapore169608
14University Hospital Vall d´HebronBarcelonaSpain08035
15Hospital Universitario La FeValenciaSpain46009

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 TitleNiCord®
Arm/Group DescriptionNiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
Period Title: Overall Study
STARTED36
COMPLETED36
NOT COMPLETED0

Baseline Characteristics

Arm/Group TitleNiCord®
Arm/Group DescriptionNiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood (UCB) cells. NiCord®
Overall Participants36
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
TitleEngraftment
DescriptionAssess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Time Frame42 days

Outcome Measure Data

Analysis Population Description
[Not Specified]
Arm/Group TitleNiCord®
Arm/Group DescriptionNiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
Measure Participants36
Number [percentage of patients]
94

Adverse Events

Time Frameone year
Adverse Event Reporting Description
Arm/Group TitleNiCord®
Arm/Group DescriptionNiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord®
All Cause Mortality
NiCord®
Affected / at Risk (%)# Events
Total16/36 (44.4%)
Serious Adverse Events
NiCord®
Affected / at Risk (%)# Events
Total34/36 (94.4%)
Immune system disorders
GvHD11/36 (30.6%)
Infections and infestations
Infection17/36 (47.2%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hematologic Malignancy8/36 (22.2%)
Other (Not Including Serious) Adverse Events
NiCord®
Affected / at Risk (%)# Events
Total36/36 (100%)
Immune system disorders
GvHD6/36 (16.7%)
Metabolism and nutrition disorders
Decreased appetite9/36 (25%)
Hyperglycemia6/36 (16.7%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukemia recurrent5/36 (13.9%)
Vascular disorders
Hypertension13/36 (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/TitleKelly Myers
OrganizationGamida Cell
Phone026595631
Emailkelly@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