The objective of this study is to make T-cell depleted stem cells from a family memberwho is a half match (haplo-identical) available on an expanded access basis to patientsreceiving one or two unrelated cord blood transplants who are at a higher risk of notengrafting in a safe amount of time. The purpose of the related stem cells is the givethe bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells willgrow and permanently rescue the bone marrow.
The primary purpose of the study is to provide expanded access of T-cell depleted
haplo-identical stem cells for patients receiving allogeneic transplantation from a
related haplo-identical donor and an unrelated, umbilical cord blood (UUCB) unit(s) for
the treatment of high risk malignancies and non-malignant disorders. The T-cell depleted
haplo-identical stems cells are intended to facilitate early, short-term myeloid
engraftment with the primary goal of minimizing early infections and other non-relapse
mortality while the UUCB cells engraft as the durable and permanent graft. Patients with
high risk or refractory malignancies, or non-malignant disorders amenable to stem cell
transplantation therapy but lacking conventional related or unrelated donors will be
eligible for this protocol.
Biological: CliniMACS CD34 Reagent System
The CliniMACS CD34 Reagent System is a medical device that is used in vitro to select and
enrich CD34+ cells from heterogeneous hematologic cell populations for transplantation in
cases where this is clinically indicated.
Inclusion Criteria:
- Have a consenting related haplo-identical (3/6, 4/6, or 5/6 if DRB1 mismatch) stem
cell donor.
- Have one or two available 4, 5, or 6/6 antigen matching unrelated UCB unit(s) that
will deliver a total cell dose >3.0 x 10e7 cells/kg. Patients who do not have a
single UCB unit that will deliver the minimum required cell dose, two partially
HLA-matched UCB units which together meet the minimum cell dose requirement, can be
used for 1 transplant. These units must be HLA-matched minimally at 4 of 6 HLA-A and
B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by
molecular typing) loci with the patient, and HLA-matched at 3 of 6 HLA- A, B, DRB1
loci with each other (using same resolution of HLA typing as indicated above). There
is no limitation on maximum cell dose.
- Have a high risk or refractory malignancy, or non-malignant disorder amenable to
stem cell transplantation therapy.
- Meet eligibility requirements for allogeneic transplant per institutional standard
practices.
- Have given written informed consent according to FDA guidelines (or consent of
parent/legal guardian as applicable).
- Be <65 years of age at the time of study enrollment.
Exclusion Criteria:
- Have a consenting 8/8 or 10/10 allele matched, consenting, related or unrelated
hematopoietic stem cell transplant (HSCT) donor.
- Have a life expectancy of less than 3 months.
- Have uncontrolled infections at time of cytoreduction.
Duke University Medical Center
Durham, North Carolina, United States
Investigator: Erin Arbuckle
erin.arbuckle@duke.edu
Erin Arbuckle
919-684-3293
erin.arbuckle@duke.edu
Joanne Kurtzberg, MD, Principal Investigator
Duke University