Umbilical Cord Transplantation for the Elderly Population
While cord blood transplants have been performed safely in elderly patients, many still relapse. The investigators propose to intensify the preparative regimen for this patient group in an attempt to decrease relapses, and combine this with an ex vivo expanded Umbilical Cord Blood (UCB) unit.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label,Double Arm,Single Center Pilot Study to Evaluate the Safety and Efficacy of Transplantation of Either StemEx, Umbilical Cord Blood Stem and Progenitor Cells Expanded ex Vivo, or an Unmanipulated Cord Blood Unit in the Elderly Population With Hematologic Malignancies Using Reduced Intensity Regimen|
- Efficacy of StemEx [ Time Frame: 100 days ] [ Designated as safety issue: No ]The primary endpoint is to demonstrate the efficacy of StemEx® vs. unmanipulated UCB transplantation in the elderly population (>55years of age) following RIC regimen by demonstrating engraftment with full donor chimerism (>98%) by Day 100 of more than 60% of the patients who received transplants expanded by the StemEx method.
- Time to engraftment [ Time Frame: 42 days ] [ Designated as safety issue: No ]The day of neutrophil engraftment is defined as the first day of 3 consecutive days of an ANC greater than 500/microliter. The platelet recovery is the first of 3 consecutive measurements tested on different days of a platelet count greater than or equal to 20,000 without requiring platelet transfusions in the previous 7 days. Patients will be monitored for donor cell engraftment as evidenced by neutrophil recovery and donor chimerism in the marrow and/or peripheral blood at serial time points post transplant.
|Study Start Date:||January 2010|
|Estimated Study Completion Date:||January 2015|
|Estimated Primary Completion Date:||January 2015 (Final data collection date for primary outcome measure)|
No Intervention: Unmanipulated arm
Participants that do not meet criteria for StemEx®, will be registered into the unmanipulated UCB arm and receive the standard conditioning regimen.
Experimental: Stemx Arm
StemEx is a stem/progenitor cell-based product of ex-vivo expanded allogeneic UCB, which is administered to the subject in combination with the non-manipulated portion of the same cord blood unit (CBU). The CBU must be cryopreserved in two portions of which the larger (or equal) CBU portion contains at least 1.5 x 107 total nucleated cells (TNC)/Kg. This portion remains unmanipulated and is transplanted on Day 0. StemEx is derived from the smaller (or equal) CBU portion, which is expanded ex vivo for 21 days starting pre-transplant in the presence of cytokines TPO, IL-6, Flt-3L and SCF at a concentration of 50ng/ml and 5μM tetraethylenepentamine (TEPA)
For patients allocated to StemEx® arm:
Allogeneic stem cell transplantation is a life saving procedure in selected high-risk or recurrent hematologic malignancies and marrow failure syndromes. However its wide application is limited by availability of suitably HLA matched adult donors. Umbilical Cord Blood (UCB) has been increasingly used as an alternative hematopoietic stem cell source for these patients. To date, over 10,000 UCB transplants have been performed in both children32-38 and adults.35,39-44 Its advantages include easier procurement, decreased risk to donors, reduced risk of transmitting infections, the immediate availability of cryopreserved units, and acceptable HLA mismatches. The transplantation of UCB allows a greater degree of HLA mismatching without an unacceptably high incidence of graft versus host disease (GVHD). Adult patients receiving myeloablative cord blood transplants have a 90% chance of engraftment, but carry a 50% rate of transplant related mortality.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01484470
|United States, Illinois|
|Loyola University Medical Center|
|Maywood, Illinois, United States, 60153|
|Principal Investigator:||Patrick Stiff, MD||Loyola Universtiy Medical Center|