Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

NCT03145545 | Unknown

• 1 other identifier: 16-013527
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective 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 Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.

Conditions

Immunodeficiencies; Leukemia; Bone Marrow Failure Syndrome

Interventions

Apha/beta T and CD19+ cell depletion using CliniMACS device DEVICE

Stem cells will be processed using the CliniMACS device for alpha/beta and CD19+ T cell depletion. Processing of cells using the CliniMACS will occur in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique.

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed, informed consent
• Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
• Patients who do not meet criteria for current open, institutional protocols using CliniMACs device for β T/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
• Sickle cell disease or thalassemia
• Other diseases treated with HSCT
• o Malignant diseases
• Acute leukemias
• Chronic leukemias
• Lymphomas
• Myelodyplastic syndrome

You may not qualify if:

• Suitable and available HLA matched sibling donor. However, patients with fully matched related donors (including siblings) may be eligible in special circumstances where use of an unmanipulated bone marrow graft has increased risks for donor or recipient.
• Donor unable to donate peripheral stem cells
• Pregnant participants
• Donor Eligibility 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 mismatched at one to five HLA alleles (haploidentical)
• Matched related donor may be considered suitable donors for this protocol if a peripheral stem cell donation is deemed by the clinical team to be a safer donation option (if donor is not a suitable candidate for a bone marrow harvest) or if there is concern that bone marrow harvest would not yield adequate cell doses Additionally, a matched related donor would be considered suitable for this protocol if there are safety concerns regarding a patient receiving a standard T cell replete bone marrow transplant due to individualized GVHD risk or risk related to standard GVHD prevention medications.
• Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, HepB, HepC PCR negative.
• We assess donor eligibility as per our Allogenic Donor Evaluation for Eligibility standard operating procedure. These 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.
• Related donors will be consented and enrolled under IRB approved research protocol for cell collection per IRB 04-004078 CHP 784 Clinical and Research Collection and Future Research Use of Bone Marrow, Stem Cells or T Cells.
• 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 IDE 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

AVAILABLE

Related Publications (27)

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MeSH Terms

Conditions

Leukemia; Bone Marrow Failure Disorders; Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Immune System Diseases

Study Officials

• Tim Olson, MD, PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Megan Atkinson

CONTACT

215-590-2820; cttsbmtintake@chop.edu

Patricia Hankins, BSN, RN

CONTACT

215-590-5168; hankinsp@chop.edu

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Hematopoietic Stem Cell Transplantation (HSCT) Program

Study Record Dates

• First Submitted: May 5, 2017
• First Posted: May 9, 2017
• Last Updated: July 8, 2026

Record last verified: 2026-07

Locations

US (1)