Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
Condition: Hematological Malignancies; Acute Lymphoblastic Leukemia (ALL); Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)
MeSH terms: Leukemia; Leukemia, Myeloid, Acute; Neoplasms; Preleukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Hematologic Neoplasms; Myelodysplastic Syndromes
Study type: Interventional
Phase: Phase 1/Phase 2

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.
    

Intervention name(s): NiCord®
Intervention type(s): Drug
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment