Clinical trial NCT01881334: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
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
Study type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Hematologic Malignancies
Inborn Errors of Metabolism Disorders
Immune Deficiencies
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
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: Duke University
name: Miltenyi Biotec, Inc.
First posted: 2013-06-19
Sex: ALL
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

Brief summary: The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is the give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow 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.

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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Joanne Kurtzberg, MD
role: PRINCIPAL_INVESTIGATOR
References: