Don't get left behind! The modernized ClinicalTrials.gov is coming. Check it out now.
Say goodbye to ClinicalTrials.gov!
The new site is coming soon - go to the modernized ClinicalTrials.gov
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01881334
Expanded Access Status : Available
First Posted : June 19, 2013
Last Update Posted : November 15, 2022
Sponsor:
Collaborators:
Duke University
Miltenyi Biotec, Inc.
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Study Description
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information
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.

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

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.
Study Design
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : 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 the National Library of Medicine


Interventions
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

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.
Eligibility Criteria
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01881334


Contacts
Layout table for location contacts
Contact: Erin Arbuckle 919-684-3293 erin.arbuckle@duke.edu

Locations
Layout table for location information
United States, North Carolina
Duke University Medical Center Available
Durham, North Carolina, United States, 27710
Contact: Erin Arbuckle       erin.arbuckle@duke.edu   
Principal Investigator: Joanne Kurtzberg, MD         
Sponsors and Collaborators
Joanne Kurtzberg, MD
Duke University
Miltenyi Biotec, Inc.
Investigators
Layout table for investigator information
Principal Investigator: Joanne Kurtzberg, MD Duke University
More Information
Go to 
Top of Page Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Joanne Kurtzberg, MD, MD, Duke University
ClinicalTrials.gov Identifier: NCT01881334    
Other Study ID Numbers: Pro00045700
First Posted: June 19, 2013    Key Record Dates
Last Update Posted: November 15, 2022
Last Verified: November 2022
Keywords provided by Joanne Kurtzberg, MD, 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
ALL
AML
MDS
 CGD
SCID
Adrenoleukodystrophy
Metachromaticleukodystrophy
Krabbe
PMD
Hunter's
Hurler's
Severe Aplastic Anemia
Lymphoma
Sickle Cell Disease
Thalassemia
Additional relevant MeSH terms:
Layout table for MeSH terms
Neoplasms
Hematologic Neoplasms
Metabolism, Inborn Errors
Metabolic Diseases
Immunologic Deficiency Syndromes
 Neoplasms by Site
Hematologic Diseases
Immune System Diseases
Genetic Diseases, Inborn