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

NCT ID: NCT01881334

Last Updated: 2025-11-20

Basic Information

• Recruitment Status: AVAILABLE
• Study Classification: EXPANDED_ACCESS

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 to 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.

Conditions

Hematologic Malignancies; Inborn Errors of Metabolism Disorders; Immune Deficiencies

Interventions

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.

Intervention Type: BIOLOGICAL

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.

• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Duke University

OTHER

Sponsor Role: collaborator

Miltenyi Biotec, Inc.

INDUSTRY

Sponsor Role: collaborator

Joanne Kurtzberg, MD

OTHER

Sponsor Role: lead

Responsible Party

Joanne Kurtzberg, MD

MD

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Joanne Kurtzberg, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

Duke University Medical Center

Durham, North Carolina, United States

Site Status: AVAILABLE

Countries

United States

Central Contacts

Erin Arbuckle

Role: CONTACT

erin.arbuckle@duke.edu

919-684-3293

Facility Contacts

Erin Arbuckle

Role: primary

erin.arbuckle@duke.edu

Other Identifiers

Pro00045700

Identifier Type: -

Identifier Source: org_study_id