Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Condition: Sickle Cell Disease & Thalassemia
MeSH terms: Anemia, Sickle Cell; Thalassemia; Hemoglobinopathies
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo
      Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
    

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.

      In Part 1 of this study, NiCord® will be administered to the patient in conjunction with a
      second, unmanipulated CBU. In Part 2 of this study, NiCord® will be administered to the
      patient without a second, unmanipulated CBU. The study duration per patient is approximately
      270 days from signing of informed consent to last visit on day 180 post-transplant.

      The overall study objectives of part 1 of this study are to evaluate the safety and efficacy
      of co-transplantation of NiCord® and an unmanipulated CBU in patients with Hemoglobinopathies
      (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy. The
      overall study objectives of part 2 of this study are to evaluate the safety and efficacy of
      transplantation of NiCord® in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or
      thalassemia major) following myeloablative therapy.

      The study hypothesis for part 1 of this study is that the co-transplantation of NiCord® and
      an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies (SCD, or
      thalassemia major) following myeloablative preparative therapy will be safe and will enable
      cord blood engraftment. The study hypothesis for part 2 of this study is that transplantation
      of NiCord® in patients with hemoglobinopathies (SCD, or thalassemia major) following
      myeloablative preparative therapy will be safe and will enable cord blood engraftment.

      Up to fifteen (15) evaluable patients recruited for part 1 of the study and up to five (5)
      patients for part 2 of the study should be 2-45 years of age, at least 10 kg in weight, have
      symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic
      myeloablative HSCT for the treatment of SCD.
    

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: Sickle Cell Disease & Thalassemia; Genetic disorder