Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

  • End date
    Jan 17, 2025
  • participants needed
  • sponsor
    Children's Hospital of Philadelphia
Updated on 17 November 2021
stem cell transplantation
graft versus host disease
acute leukemia
shortening fraction
bone marrow failure
chronic leukemia
allogeneic hematopoietic stem cell transplant
chronic leukemias
acute leukemias


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.


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.

Condition Leukemia, Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome, Immune Deficiency, Immunodeficiency, immunodeficiencies, Inborn error of metabolism, inborn errors of metabolism, decreased immune function, Leukemia (Pediatric), Primary Immunodeficiency Disorders, leukemia, Metabolic disorder, Metabolic Disorders, leukemias, immunodeficient
Treatment Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Clinical Study IdentifierNCT02356653
SponsorChildren's Hospital of Philadelphia
Last Modified on17 November 2021


Yes No Not Sure

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
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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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