This phase I trial studies the effects of valproic acid expanded umbilical cord blood stem cells in treating adult patients with hematological malignancies undergoing donor stem cell transplant. Expanding or growing umbilical cord blood stem cells in a laboratory using valproic acid may lead to faster white blood cell count recovery, lower the risk of infections, and improve transplant results compared to umbilical cord blood stem cells that have not been expanded.
Additional locations may be listed on ClinicalTrials.gov for NCT03885947.
See trial information on ClinicalTrials.gov for a list of participating sites.
PRIMARY OBJECTIVES:
I. To evaluate the safety of the valproic acid-expanded umbilical cord blood-derived CD34-positive cells valproic acid (VPA) expanded cord blood stem cells as determined by the incidence of infusion related reactions and graft failure in patients with hematological malignancies undergoing allogeneic hematopoietic cell transplantation (HCT).
SECONDARY AND EXPLORATORY OBJECTIVES:
I. Evaluate time to neutrophil and platelets engraftment.
II. Evaluate transplant-related mortality (TRM).
III. Evaluate disease free and overall survivals.
IV. Evaluate risk of graft versus host disease (GVHD)
V. Evaluate incidence of infectious complications.
VI. Assess the kinetics of engraftment and immune reconstitution.
OUTLINE:
CONDITIONING REGIMEN: All patients receive cyclophosphamide intravenously (IV) over 2 hours on day -6, fludarabine IV over 30 minutes on days -6 to -2, thiotepa IV over 2 hours on days -5 and -4, and total body irradiation (TBI) on days -2 and -1.
UMBILICAL CORD BLOOD (UCB) INFUSION: There would be 2 consecutive cohorts. First 5-7 patients will be assigned to cohort I. Upon successful enrollment of the first cohort patients will be assigned to cohort II.
COHORT I: Patients receive a unit of valproic acid-expanded UCB-derived CD34-positive cells, a unit of the T-cell containing portion of the expanded UCB, and a unit of UCB that has not been manipulated, all via infusion. All UCB unit infusions occur on day 0 and are separated by at least 2 hours.
COHORT II: Patients receive a unit of valproic acid-expanded UCB-derived CD34-positive cells and a unit of the T-cell containing portion of the expanded UCB as in Cohort I. Both parts of UCB unit infusions occur on day 0 and are separated by at least 2 hours.
GVHD PROPHYLAXIS: All patients receive tacrolimus IV continuously every 24 hours or orally (PO) on days -3 to 120 and mycophenolate mofetil IV or PO three times daily (TID) on days 0-60.
After completion of study treatment, patients are followed up to 1 year.
Lead OrganizationIcahn School of Medicine at Mount Sinai
Principal InvestigatorAlla Keyzner
