Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Recruiting now · Early Phase 1

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

In brief

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.

Key facts

Study ID: NCT02356653
Run by: Children's Hospital of Philadelphia
People needed: 100
Starts: 2013-12-01
Expected to finish: 2030-01-01
Last updated by the study team: 2026-02-20

Who can join

Age: any, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but are not deemed suitable candidates per their treating clinical team 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/including HLH, Hemoglobinopathies correctable and requiring HSCT, and Other diseases treated with HSCT/Other non-malignant blood, metabolic, or immune disorders for which HSCT has been recommended
Malignant diseases:
Acute leukemias, Chronic leukemias, Lymphomas, Myelodyplastic syndrome
Signed informed consent
Lansky or Karnofsky performance ≥60
Hematologic and Organ Function per current institutional SOP.
Infectious Evaluation as per current institutional SOP.
Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.
Patients must have an identified living donor
Donor selection will comply with 21 CFR 1271
Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10/10 match, a 9/10 match, or an 8/10 match if one of the mismatches is at DQB1
Related donor suitable for mobilization infectious disease criteria as per SOP, including HIV, HepB, HepC PCR negative.
CHOP BMT procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is FACT accredited.
Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis.
The donors selected for this IND will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases

You may not qualify if…

Uncontrolled bacterial, viral or fungal infections
Suitable, fully HLA matched sibling donor
Donor unable to donate peripheral stem cells
Pregnant participants

Where it is running

Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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