Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Study Overview
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Brief Summary
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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
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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.

Official Title
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Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells

Conditions
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Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

Intervention / Treatment
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* Device: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 30 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional 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)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Expanded access to CliniMACs device for T cell depletion<br>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. | Device: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)<br>* 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.<br>* Other names: CliniMACs;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival |  | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Graft versus Host Disease |  | 1 year | 
| Graft Failure |  | 100 days |