Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy
Condition: Hematologic Malignancies; Acute Myeloid Leukemia; Lymphoid Leukemia; Chronic Myeloid Leukemia; Hodgkin's Disease; Non-Hodgkin's Lymphoma; Myelodysplastic Syndromes
MeSH terms: Leukemia; Leukemia, Myeloid; Neoplasms; Hematologic Neoplasms; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Hodgkin Disease; Leukemia, Lymphoid; Myelodysplastic Syndromes
Study type: Interventional
Phase: Phase 2/Phase 3

Summary: 
      The purpose of this study is to determine the efficacy and safety of transplanting StemEx® in
      patients with certain hematological malignancies. For these patients, it is suggested that
      StemEx® can improve upon the outcome of transplanting a single, unmanipulated cord blood unit
      by significantly increasing the number of stem/progenitor cells available to the patient.
    

Detailed description: 
      Allogeneic hematopoietic stem cell transplantation is a life-saving procedure for patients
      with hematologic malignancies; yet wide application of this procedure is limited by the
      availability of suitably Human Leukocyte Antigen (HLA) - matched donors. Only 30% of patients
      who could benefit from this procedure have an HLA-matched sibling. The lengthy search for a
      matched donor may critically delay transplantation. In addition, far fewer patients of racial
      minorities find suitable HLA-matched donors. Umbilical cord blood (UCB) has been increasingly
      used as an alternative source of stem cells; however, its use in adults and adolescent
      patients is limited due to insufficient cell dose required for satisfactory hematopoietic
      reconstitution.

      Gamida Cell - Teva Joint Venture Ltd. is engaged in the development of StemEx®, an expanded
      hematopoietic UCB stem cell graft, as a potential medicinal product for the treatment of
      cancer and hematological malignancies. The expansion technology enables preferential
      expansion of hematopoietic stem and early progenitor cells and is based on the findings that
      copper chelators can regulate the balance between self-renewal and differentiation of stem
      cells.

      The multi-national, multi-center Phase II/III clinical study designated to evaluate the
      safety and efficacy of StemEx® will enroll approximately 100 subjects with high-risk
      hematologic malignancies who are candidates for allogeneic stem cell transplantation (SCT).
      This study will evaluate the effect of StemEx® on overall survival as measured by overall
      100-day mortality.

      The study consists of 4 phases:

        1. Screening phase includes subjects' clinical assessment and screening tests

        2. Conditioning phase includes the myeloablative treatment prior transplantation procedure

        3. Transplantation and post-transplant follow-up phase to day 180

        4. Observational phase: survival status follow-up to day 730 (18 months)
    

Intervention name(s): StemEx®
Intervention type(s): Drug
Intervention description(s): The stem/progenitor cell based product composed of ex vivo expanded allogeneic umbilical cord blood cells, which is infused to subject at a rate of 1-3 ml/min in combination with non-manipulated cells derived from the same cord blood unit.
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Tetraethylenepentamine; Umbilical Cord Blood Stem Cell Transplantation; Hematological Malignancies; Acute Lymphoid Leukemia; Subjects with high-risk hematologic malignancies who are candidates for allogeneic stem cell transplantation