Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Hematological Malignancies, Myelodysplastic Syndrome (MDS)
Conditions
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.
Interventions
DRUGNiCord®
Sponsors
Gamida Cell ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 65 Years
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Engraftment | 42 days | Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation. |
Countries
Italy, Netherlands, Singapore, Spain, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| NiCord® NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood (UCB) cells.
NiCord® | 36 |
| Total | 36 |
Baseline characteristics
| Characteristic | NiCord® |
|---|---|
| Adjusted disease risk index High | 13 Participants |
| Adjusted disease risk index Intermediate | 15 Participants |
| Adjusted disease risk index Low | 8 Participants |
| Age, Continuous | 44 Years |
| Primary diagnosis Acute lymphoblastic leukemia (ALL) | 9 Participants |
| Primary diagnosis Adult acute myeloid leukemia (AML) | 17 Participants |
| Primary diagnosis Chronic myeloid leukemia (CML) | 2 Participants |
| Primary diagnosis Hodgkins Disease | 1 Participants |
| Primary diagnosis Myelodysplastic syndromes (MDS) | 7 Participants |
| Primary diagnosis non-Hodgkins Lymphoma | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 30 Participants |
| Sex: Female, Male Female | 16 Participants |
| Sex: Female, Male Male | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 16 / 36 |
| other Total, other adverse events | 36 / 36 |
| serious Total, serious adverse events | 34 / 36 |
Outcome results
Primary
Engraftment
Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Time frame: 42 days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| NiCord® | Engraftment | 94 percentage of patients |