Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
Phase 1/Phase 2 |
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
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
- Engraftment [42 days]
Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Eligibility Criteria
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
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.- GC P#03.01.020
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
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
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 |
kelly@gamida-cell.com |
- GC P#03.01.020