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Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC (ExpMACs)

Primary Purpose

Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes

Status
Recruiting
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Sponsored by
Children's Hospital of Philadelphia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leukemia

Eligibility Criteria

undefined - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.

  2. Patients with the following transplantable diseases:

    Non-malignant diseases:

    Metabolic storage diseases correctable by HSCT

    Bone marrow failure syndromes

    Immunodeficiencies/immune dysregulation syndromes

    Malignant diseases:

    Acute leukemias

    Chronic leukemias

    Lymphomas

    Myelodyplastic syndrome

  3. Organ function criteria:

    Lansky or Karnofsky performance ≥60

    Serum creatinine ≤3xupper limit of normal for age

    Hepatic: Transaminases ≤10xnormal

    Cardiac shortening fraction ≥27%

    Bilirubin <2.5x normal (unless elevation due to Gilberts disease)

  4. No active untreated infection
  5. Signed informed consent
  6. No fully HLA matched sibling donor available
  7. Females of childbearing potential must have negative pregnancy test

Exclusion Criteria:

  1. Uncontrolled bacterial, viral or fungal infections
  2. Fully HLA matched sibling donor
  3. Donor unable to donate peripheral stem cells
  4. Pregnant Females

Sites / Locations

  • Children's Hospital of PhiladelphiaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Expanded access to CliniMACs device for T cell depletion

Arm Description

access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion.

Outcomes

Primary Outcome Measures

Overall Survival

Secondary Outcome Measures

Graft versus Host Disease
Graft Failure

Full Information

First Posted
October 22, 2014
Last Updated
February 20, 2023
Sponsor
Children's Hospital of Philadelphia
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1. Study Identification

Unique Protocol Identification Number
NCT02356653
Brief Title
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Acronym
ExpMACs
Official Title
Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 2013 (undefined)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
January 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Philadelphia

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
Detailed Description
Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Model Description
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Expanded access to CliniMACs device for T cell depletion
Arm Type
Experimental
Arm Description
access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion.
Intervention Type
Device
Intervention Name(s)
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Other Intervention Name(s)
CliniMACs
Intervention Description
Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
Primary Outcome Measure Information:
Title
Overall Survival
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Graft versus Host Disease
Time Frame
1 year
Title
Graft Failure
Time Frame
100 days

10. Eligibility

Sex
All
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion. Patients with the following transplantable diseases: Non-malignant diseases: Metabolic storage diseases correctable by HSCT Bone marrow failure syndromes Immunodeficiencies/immune dysregulation syndromes Malignant diseases: Acute leukemias Chronic leukemias Lymphomas Myelodyplastic syndrome Organ function criteria: Lansky or Karnofsky performance ≥60 Serum creatinine ≤3xupper limit of normal for age Hepatic: Transaminases ≤10xnormal Cardiac shortening fraction ≥27% Bilirubin <2.5x normal (unless elevation due to Gilberts disease) No active untreated infection Signed informed consent No fully HLA matched sibling donor available Females of childbearing potential must have negative pregnancy test Exclusion Criteria: Uncontrolled bacterial, viral or fungal infections Fully HLA matched sibling donor Donor unable to donate peripheral stem cells Pregnant Females
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Margaret Tartaglione, RN
Phone
215-590-4029
Email
tartaglione@chop.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Patricia Hankins, RN
Phone
215-590-5168
Email
hankinsp@chop.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nancy J Bunin, MD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Margie Tartaglione, RN
Phone
215-590-4029
Email
tartaglione@chop.edu
First Name & Middle Initial & Last Name & Degree
Patricia Hankins, RN
Phone
215-590-5168
Email
hankinsp@chop.edu
First Name & Middle Initial & Last Name & Degree
Nancy J Bunin, MD
First Name & Middle Initial & Last Name & Degree
Stephan A Grupp, MD, PhD

12. IPD Sharing Statement

Learn more about this trial

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

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