Study of Allogeneic Umbilical Cord Blood Infusion for Adults With Ischemic Stroke
Condition: Stroke; Stroke, Acute; Brain Injury, Acute
MeSH terms: Stroke; Brain Injuries; Ischemic Stroke
Study type: Interventional
Phase: Phase 2

Summary: 
      The primary objective of this study is to determine the efficacy of a single intravenous
      infusion of unrelated donor umbilical cord blood (UCB) for improving functional outcomes in
      patients with ischemic stroke. Eligible subjects will receive an intravenous infusion of UCB
      or placebo 3-10 days following stroke. Subjects will not receive immunosuppressive or
      myeloablative medications prior to the infusion. Subjects will be followed for one year post
      infusion for safety and efficacy. Assessments will examine safety and tolerability of the
      infusion, change in neurological symptoms, change in quality of life, and emotional and
      cognitive status. Assessments will occur at 24 hours post infusion, and at 30, 90, 180 and
      365 days post infusion.
    

Detailed description: 
      This is a multicenter, placebo controlled, randomized, double blinded Phase 2 study in 100
      subjects 18-90 years of age who have sustained a recent ischemic stroke. Potential subjects
      can be screened and consented the day of their stroke (Day 1). Treatment with umbilical cord
      blood (UCB) cells or placebo will be administered intravenously as a single infusion as early
      as 3 days but no later than 10 days after the patient's stroke. UCB units will be selected
      from an accredited U.S. public cord bank based on blood type, race and a targeted cell dose
      ranging between 0.5 to 5 x 10^7 total nucleated cell count (TNCC)/kg. Study subjects will not
      receive immunosuppressive or myeloablative medications prior to infusion of the cord blood or
      placebo.

      All subjects, families and medical staff will be blinded to treatment arm. When a subject is
      randomized to study drug at a clinical site without a cord blood bank, the selected cord
      blood units (CBU) will be shipped frozen overnight to the site. Once selected and available
      on site, each CBU will be thawed, washed, tested, released and infused intravenously using
      common standard operating procedures (SOPs) at all sites. For subjects randomized to placebo,
      a diluent with the same appearance and odor as a CBU will be prepared.

      Patients will have baseline magnetic resonance imaging (MRI) and will be assessed at 1, 3, 6,
      and 12 months for functional outcomes. All patients will receive standard of care therapy
      while enrolled in this study and all subjects will be strongly encouraged to participate in
      rehabilitative therapy.

      The primary objective of the study is to determine, in a randomized, placebo controlled
      trial, the efficacy of a single intravenous (IV) infusion of unrelated donor UCB for
      improving functional outcomes in patients with ischemic stroke. The secondary objectives are
      as follows:

        1. To describe the safety and tolerability of a single IV infusion of unrelated donor UCB
           in patients with ischemic stroke

        2. To evaluate the efficacy of a single IV infusion of unrelated donor UCB for improvement
           of neurological symptoms following ischemic stroke

        3. To evaluate the efficacy of a single IV infusion of unrelated donor UCB for improvement
           in quality of life and emotional and cognitive status in patients with ischemic stroke
    

Intervention name(s): Umbilical Cord Blood; Placebo
Intervention type(s): Biological; Other
Intervention description(s): Umbilical cord blood will be infused intravenously through a peripheral IV line after premedication with diphenhydramine, hydrocortisone, and acetaminophen. Units will be from a public cord blood bank with selection based on blood type, race, and the number of cells in the pre cryopreservation product, targeting a dose range of 0.5 to 5 x 10^7 TNCC/kg.; The placebo product will be acellular and will consist of tissue culture medium 199 (TC-199 [pink]) with 1% dimethyl sulfoxide (DMSO), which are standard components in cellular products. The volume of placebo product will be 50 mL, which is in the range of a typical UCB unit that has been washed and thawed after cryopreservation. Infusion and premedication procedures will be the same as those conducted for the umbilical cord blood arm.
Other intervention name(s): cord blood; hematopoietic stem cells
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: hematopoietic stem cells; umbilical cord blood; stroke; ischemic stroke