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

June 13, 2024 updated by: Timothy Olson, Children's Hospital of Philadelphia

Expanded Access Protocol Using TCR Alpha/Beta T Cell/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

Only 25-30% of patients who may benefit from HSCT have a matched related donor. An unrelated cord blood may not be available due to size or matching criteria, or if a reduced intensity regiment is recommended. The risk of severe graft vs. host disease (GVHD) and other complications is higher with unrelated donors, or partially matched related donors. At the Children's Hospital of Philadelphia (CHOP) there is extensive experience using mismatched unrelated donors or partially matched related donors with complete or partial T depletion to reduce the risk of severe GVHD.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Available
        • Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

PATIENT AND DONOR ELIGIBILITY

Patients who lack an 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 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

Malignant diseases:

  • Acute leukemias
  • Chronic leukemias
  • Lymphomas
  • Myelodyplastic syndrome

Organ function criteria:

It is important to note that the conditioning prescribed to the patient will be determined based on the disease and organ status and will be regimens considered standard. Appropriate combinations of chemotherapy, immunotherapy and/or radiation will be determined on an individual basis.

Patient eligibility will be assessed as per our institutional standard operating procedures:

  • Lansky or Karnofsky performance >60
  • Renal function: will be determined based on serum creatinine as per our Institutional SOP
  • Hepatic: Transaminases will be assessed as per current institutional SOP
  • Cardiac: Cardiac function will be assessed as per institutional SOP
  • No active untreated infection
  • Signed informed consent
  • No fully HLA matched sibling donor available.
  • Females of childbearing potential must have negative pregnancy test.
  • Subjects with graft failure who require a second HSCT will not need to meet eligibility criteria again prior to the second transplant. Graft failure is a medical emergency that requires HSCT

Donor Eligibility Patients must have an identified living donor

  • Donor selection will comply with 21 Code of Federal Regulations (CFR) 1271*
  • Unrelated donor that meets the matching criteria of the NMDP: Unrelated donors that may be up to a one antigen mismatch at A, B or DRB1. donor
  • Related donor mismatched at one to five antigens (haploidentical)
  • Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, Hepatitis B (HepB), Hepatitis C (HepC) polymerase chain reaction (PCR) negative.
  • CHOP bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is Foundation for the Accreditation of Cellular Therapy (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 investigational new drug (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:

  • Uncontrolled bacterial, viral or fungal infections
  • Fully HLA matched sibling donor
  • Donor unable to donate peripheral stem cells
  • Pregnant Females

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital of Philadelphia

Investigators

  • Principal Investigator: Tim Olson, MD, PhD, Children's Hospital of Philadelphia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

May 5, 2017

First Submitted That Met QC Criteria

May 5, 2017

First Posted (Actual)

May 9, 2017

Study Record Updates

Last Update Posted (Actual)

June 14, 2024

Last Update Submitted That Met QC Criteria

June 13, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 16-013527

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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