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 February 2016 by Duke University
Duke University
Miltenyi Biotec, Inc.
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University Medical Center
ClinicalTrials.gov Identifier:
First received: June 17, 2013
Last updated: February 4, 2016
Last verified: February 2016
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, 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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, MD
Duke University
Miltenyi Biotec, Inc.
Principal Investigator: Joanne Kurtzberg, MD Duke University
  More Information

Responsible Party: Joanne Kurtzberg, MD, 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: February 4, 2016
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:
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
Severe Aplastic Anemia
Sickle Cell Disease

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Immune System Diseases

ClinicalTrials.gov processed this record on May 26, 2016