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

Primary Purpose

Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies

Status

Available

Phase

Locations

United States

Study Type

Expanded Access

Intervention

CliniMACS CD34 Reagent System

About this trial

This is an expanded access trial for Hematologic Malignancies focused on measuring 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

Eligibility Criteria

undefined - 65 Years (Child, Adult, Older Adult)All Sexes

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.

Sites / Locations

Duke University Medical Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT01881334

First Posted

June 17, 2013

Last Updated

November 14, 2022

Sponsor

Joanne Kurtzberg, MD

Collaborators

Duke University, Miltenyi Biotec, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT01881334

Brief Title

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

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

Study Type

Expanded Access

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Available

Study Start Date

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Primary Completion Date

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Study Completion Date

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3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Joanne Kurtzberg, MD

Collaborators

Duke University, Miltenyi Biotec, Inc.

4. Oversight

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies

Keywords

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

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type

Biological

Intervention Name(s)

CliniMACS CD34 Reagent System

Intervention Description

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.

10. Eligibility

Sex

All

Maximum Age & Unit of Time

65 Years

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Erin Arbuckle

Phone

919-684-3293

erin.arbuckle@duke.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Joanne Kurtzberg, MD

Organizational Affiliation

Duke University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

Individual Site Status

Available

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Erin Arbuckle

erin.arbuckle@duke.edu

First Name & Middle Initial & Last Name & Degree

Joanne Kurtzberg, MD

12. IPD Sharing Statement

Learn more about this trial

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

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