Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC (ExpMACs)

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

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 Overview

• Status: Recruiting
• Conditions: Leukemia; Bone Marrow Failure Syndromes; Immunodeficiencies; Inborn Errors of Metabolism; Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
• Intervention / Treatment: Biological: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 100
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Patricia Hankins, BSN, RN, CCRC; Phone Number: 215-590-5168; Email: hankinsp@chop.edu
• Study Contact Backup: Name: Megan Atkinson; Phone Number: 215-590-2820; Email: cttsbmtintake@chop.edu

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Sub-Investigator: Stephan A Grupp, MD, PhD
      • Principal Investigator: Timothy Olson, MD, PhD
      • Contact: Patricia Hankins, BSN, RN, CCRC; Phone Number: 215-590-5168; Email: hankinsp@chop.edu
      • Contact: Megan Atkinson; Phone Number: 215-590-2820; Email: cttsbmtintake@chop.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 30 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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.

2. 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

3. Signed informed consent
4. Lansky or Karnofsky performance ≥60
5. Hematologic and Organ Function per current institutional SOP.
6. Infectious Evaluation as per current institutional SOP.
7. Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
8. In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.

9. 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

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Expanded access to CliniMACs device for T cell depletion; 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.

Biological: 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.; Other Names: CliniMACs

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	Number of participants who remain alive.	1 year post transplant

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Graft versus Host Disease	Number of participants who had grades II-IV acute graft versus host disease and/or limited or extensive chronic gvhd	1 year post transplant
Graft Failure	Number of participants who experienced primary or secondary graft failure or autologous reconstitution.	1 year post transplant

Collaborators and Investigators

• Sponsor: Children's Hospital of Philadelphia
• Investigators: Principal Investigator: Timothy Olson, MD, PhD, Children's Hospital of Philadelphia

Study record dates

Study Major Dates

• Study Start: December 1, 2013
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2030

Study Registration Dates

• First Submitted: October 22, 2014
• First Submitted That Met QC Criteria: February 4, 2015
• First Posted (Estimated): February 5, 2015

Study Record Updates

• Last Update Posted (Actual): February 20, 2026
• Last Update Submitted That Met QC Criteria: February 18, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Genetic Diseases, Inborn; Metabolic Diseases; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Bone Marrow Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Leukemia; Immunologic Deficiency Syndromes; Metabolism, Inborn Errors; Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome
• Other Study ID Numbers: 13-010286; 12BT125 (Other Identifier: Children's Hospital of Philadelphia)

Drug and device information, study documents

• product manufactured in and exported from the U.S.: No