Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

NCT02356653 | Recruiting Early Phase 1 DMC

• 2 other identifiers: 13-01028612BT125
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Leukemia; Inborn Errors of Metabolism; Bone Marrow Failure Syndromes; Immunodeficiencies; Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

Outcome Measures

Primary Outcomes (1)

• Overall Survival

  Number of participants who remain alive.

  1 year post transplant

Secondary Outcomes (2)

• Graft versus Host Disease

  1 year post transplant

• Graft Failure

  1 year post transplant

Study Arms (1)

Expanded access to CliniMACs device for T cell depletion

EXPERIMENTAL

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)

Interventions

Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) BIOLOGICAL

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.

Also known as: CliniMACs

Expanded access to CliniMACs device for T cell depletion

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Conditions

Leukemia; Metabolism, Inborn Errors; Bone Marrow Failure Disorders; Immunologic Deficiency Syndromes; Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Bone Marrow Diseases; Immune System Diseases

Study Officials

• Timothy 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, CCRC

CONTACT

215-590-5168; hankinsp@chop.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Hematopoietic Stem Cel Transplantation (HSCT) Program

Model Details: 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.

Study Record Dates

• First Submitted: October 22, 2014
• First Posted: February 5, 2015
• Study Start: December 1, 2013
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2030
• Last Updated: February 20, 2026

Record last verified: 2026-02

Locations

US (1)