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

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ClinicalTrials.gov Identifier: NCT03145545
Expanded Access Status : Available
First Posted : May 9, 2017
Last Update Posted : January 19, 2023
Sponsor:
Information provided by (Responsible Party):
Nancy Bunin, Children's Hospital of Philadelphia

Study Description
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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.

Condition or disease Intervention/treatment
Leukemia Bone Marrow Failure Syndrome Immunodeficiencies Device: Apha/beta T and CD19+ cell depletion using CliniMACS device

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 Design
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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Expanded Access Protocol Using TCR Alpha/Beta T Cell/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Organ Donation

Interventions
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Intervention Details:
  • Device: Apha/beta T and CD19+ cell depletion using CliniMACS 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
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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03145545


Contacts
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Contact: Barbara McGlynn, BSN, RN 215-590-1303 MCGLYNN@chop.edu
Contact: Patricia Hankins, RN 215-590-5168 hankinsp@chop.edu

Locations
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United States, Pennsylvania
Children's Hospital of Philadelphia Available
Philadelphia, Pennsylvania, United States, 19104
Contact: Barbara McGlynn, BSN, RN       MCGLYNN@chop.edu   
Sponsors and Collaborators
Children's Hospital of Philadelphia
More Information
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Responsible Party: Nancy Bunin, Director, Blood and Marrow Transplant, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT03145545    
Other Study ID Numbers: 16-013527
First Posted: May 9, 2017    Key Record Dates
Last Update Posted: January 19, 2023
Last Verified: January 2023
Additional relevant MeSH terms:
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Bone Marrow Failure Disorders
Pancytopenia
Bone Marrow Diseases
Hematologic Diseases