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

Brief Title

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

Official Title

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

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.
    

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




Condition

Leukemia

Intervention

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


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device





Eligibility 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 with severe central nervous system (CNS) vasculopathy

          -  Other hemoglobinopathies requiring 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 shortening fraction >27% as per institutional SOP

          -  Bilirubin <2.5x normal (unless elevation due to Gilberts disease) 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.

        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
      

Gender

All

Ages

1 Month - N/A


Contacts

, 215-590-1303, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03145545

Organization ID

16-013527


Responsible Party

Principal Investigator

Study Sponsor

Children's Hospital of Philadelphia


Study Sponsor

, , 


Verification Date

July 2021