Philadelphia, Pennsylvania 19104


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.

Study summary:

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.


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



Primary Contact:

Principal Investigator
Nancy J Bunin, MD
Children's Hospital of Philadelphia

Margaret Tartaglione, RN
Phone: 215-590-4029

Backup Contact:

Patricia Hankins, RN
Phone: 215-590-5168

Location Contact:

Philadelphia, Pennsylvania 19104
United States

Margie Tartaglione, RN
Phone: 215-590-4029

Site Status: Recruiting

Data Source:

Date Processed: October 18, 2018

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