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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)

Intervention / Treatment
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* Drug: NiCord®


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NiCord®<br>NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. | Drug: NiCord®<br> <br> |

What is the study measuring?
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Primary Outcome Measures

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