Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies
Condition: Acute Lymphoblastic Leukemia (ALL); Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Non-Hodgkin's Lymphoma; Hodgkin's Disease
MeSH terms: Leukemia; Neoplasms; Preleukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Hematologic Neoplasms; Hodgkin Disease; Myelodysplastic Syndromes
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a
      UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second,
      Unmanipulated CBU in Patients with Hematological Malignancies
    

Detailed description: 
      Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative procedure
      for various hematological malignancies, bone marrow failure syndromes and inherited metabolic
      disorders. The application of allogeneic HSCT is limited by donor availability such that only
      approximately one-third of the otherwise appropriate candidates have suitably matched family
      donors. Alternative donors include mismatched family members or matched unrelated donors, but
      these approaches are often complicated by an increased risk of graft-versus-host disease
      (GvHD) and a prolonged and cumbersome search and procurement process. In addition, far fewer
      subjects of racial minorities find suitable human leukocyte antigen (HLA)-matched donors.

      Umbilical cord blood has been increasingly used as an alternative source of stem cells and
      has extended the availability of allogeneic HSCT to patients who would otherwise not be
      eligible for this curative approach. In the last decade the number of cord blood
      transplantations from related and unrelated donors has increased dramatically. It is
      estimated that more than 20,000 patients have undergone cord blood transplantation from
      unrelated donors to date for a variety of genetic, hematological, immunological, metabolic
      and oncologic disorders. The major advantages of cord blood transplantation include easy
      procurement, no risk to donors, reduced incidence of transmitting infections, immediate
      availability, and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch.
      Nevertheless, the low cell dose remains a main limitation of this cell source leading to
      delayed hematopoietic reconstitution, higher risk of graft failure and relatively high
      treatment related mortality rates as compared to other hematopoeitic cell sources. To improve
      outcomes and extend applicability of cord blood transplantation, one potential solution is ex
      vivo expansion of cord blood-derived stem and progenitor cells.

      The Sponsor has undertaken to develop NiCord®, which is based on a novel technology for 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 broader application of umbilical cord blood transplantation and
      improve clinical outcomes in subjects with high-risk hematological malignancies.

      The main objective of the current study is to evaluate the safety of co-transplantation of
      NiCord® and an unmanipulated cord blood unit in patients with hematological malignancies
      following myeloablative therapy.
    

Intervention name(s): NiCord®
Intervention type(s): Drug
Intervention description(s): NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Double Umbilical Cord Blood Stem Cell Transplantation; Hematological Malignancies; HLA Mismatched Donors; Cord Blood Transplantation