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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


N/A
N/A
65 Years
Open (Enrolling)
Both
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies

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Trial Information

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


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.


Inclusion Criteria:



- Have a consenting related haplo-identical (3/6 or 4/6) 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.

Type of Study:

Expanded Access

Study Design:

N/A

Principal Investigator

Joanne Kurtzberg, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University

Authority:

United States: Food and Drug Administration

Study ID:

Pro00045700

NCT ID:

NCT01881334

Start Date:

Completion Date:

Related Keywords:

  • Hematologic Malignancies
  • Inborn Errors of Metabolism Disorders
  • Immune Deficiencies
  • Haploidentical Donor
  • T-cell depleted Stem Cells
  • Allogeneic Transplant
  • Umbilical Cord Blood Donor
  • High Risk Malignancies
  • Metabolic Disorders
  • Immune Deficiency
  • Acute Lympoblastic 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
  • Neoplasms
  • Metabolic Diseases
  • Metabolism, Inborn Errors
  • Hematologic Neoplasms

Name

Location

Duke University Medical Center Durham, North Carolina  27710