Official Title
A Compassionate Release Protocol: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Allogeneic Transplantation Using a Related Haplo-Identical Donor and Unrelated, Umbilical Cord Blood Donor(s) for the Treatment of High Risk Malignancies or Non-Malignant Disorders Requiring Allogeneic Transplantation
Brief Summary

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.

Detailed Description

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.

Available
Hematologic Malignancies
Inborn Errors of Metabolism Disorders
Immune Deficiencies

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.

Eligibility Criteria

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.

Eligibility Gender
All
Eligibility Age
Minimum: N/A ~ Maximum: 65 Years
Countries
United States
Locations

Duke University Medical Center
Durham, North Carolina, United States

Investigator: Erin Arbuckle
erin.arbuckle@duke.edu

Investigator: Joanne Kurtzberg, MD

Contacts

Erin Arbuckle
919-684-3293
erin.arbuckle@duke.edu

Joanne Kurtzberg, MD, Principal Investigator
Duke University

Joanne Kurtzberg, MD
NCT Number
Keywords
Haploidentical Donor
T-cell depleted Stem Cells
Allogeneic Transplant
Umbilical Cord Blood Donor
High Risk Malignancies
Metabolic Disorders
Immune Deficiency
Acute Lymphoblastic Leukemia
Acute Myelogenous Leukemia
Myelodysplastic Syndrome
ALL
AML
MDS
CGD
SCID
Adrenoleukodystrophy
Metachromaticleukodystrophy
Krabbe
PMD
Hunter's
Hurler's
Severe Aplastic Anemia
Lymphoma
Sickle cell disease
Thalassemia
MeSH Terms
Neoplasms
Hematologic Neoplasms
Metabolism, Inborn Errors
Metabolic Diseases
Immunologic Deficiency Syndromes