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

Hematologic Malignancies Clinical Trial

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

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT01881334
• Other study ID #: Pro00045700
• Status: Available

Study information

• Verified date: November 2023
• Source: Duke University
• Contact: Erin Arbuckle
• Phone: 919-684-3293
• Email: erin.arbuckle[@]duke.edu
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: N/A to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hematologic Malignancies
• Hematologic Neoplasms
• Immune Deficiencies
• Immunologic Deficiency Syndromes
• Inborn Errors of Metabolism Disorders
• Metabolic Diseases
• Metabolism, Inborn Errors
• Neoplasms

Intervention

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.

Locations

Country	Name	City	State
United States	Duke University Medical Center	Durham	North Carolina

Sponsors (3)

Lead Sponsor	Collaborator
Joanne Kurtzberg, MD	Duke University, Miltenyi Biotec, Inc.

Country where clinical trial is conducted

United States,