Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Expanded access is currently available for this treatment.
Verified December 2013 by Duke University
Duke University
Miltenyi Biotec, Inc.
Information provided by (Responsible Party):
Joanne Kurtzberg, Duke University Medical Center
ClinicalTrials.gov Identifier:
First received: June 17, 2013
Last updated: December 23, 2013
Last verified: December 2013

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.

Condition Intervention
Hematologic Malignancies
Inborn Errors of Metabolism Disorders
Immune Deficiencies
Biological: CliniMACS CD34 Reagent System

Study Type: Expanded Access     What is Expanded Access?
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

Resource links provided by NLM:

Further study details as provided by Duke University:

Intervention Details:
    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.
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.


Ages Eligible for Study:   up to 65 Years
Genders Eligible for Study:   Both

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01881334

Contact: Maggie D Peterson, RN 919-668-1280 maggie.peterson@duke.edu
Contact: Jennifer H Baker, RN 919-668-6536 jennifer.hungate@dm.duke.edu

United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Contact: Maggie D Peterson, RN       maggie.peterson@duke.edu   
Principal Investigator: Joanne Kurtzberg, MD         
Sponsors and Collaborators
Joanne Kurtzberg
Duke University
Miltenyi Biotec, Inc.
Principal Investigator: Joanne Kurtzberg, MD Duke University
  More Information

No publications provided

Responsible Party: Joanne Kurtzberg, MD, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01881334     History of Changes
Other Study ID Numbers: Pro00045700
Study First Received: June 17, 2013
Last Updated: December 23, 2013
Health Authority: United States: Food and Drug Administration
United States: Center for International Blood and Marrow Transplant Research
United States: Institutional Review Board

Keywords provided by Duke University:
Metabolic Disorders
Sickle Cell Disease
Haploidentical Donor
T-cell depleted Stem Cells
Allogeneic Transplant
Umbilical Cord Blood Donor
High Risk Malignancies
Immune Deficiency
Acute Lympoblastic Leukemia
Acute Myelogenous Leukemia
Myelodysplastic Syndrome
Severe Aplastic Anemia

Additional relevant MeSH terms:
Metabolic Diseases
Metabolism, Inborn Errors
Hematologic Neoplasms
Genetic Diseases, Inborn
Neoplasms by Site
Hematologic Diseases

ClinicalTrials.gov processed this record on April 17, 2014