Expanded Access Protocol for Tabelecleucel in Subjects With EBV-Associated Viremia or Malignancies
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The primary objective of this protocol is to provide access to tabelecleucel to patients with Epstein-Barr virus-positive post-transplant lymphoproliferative disorder (EBV+ PTLD), EBV+ primary immunodeficiency lymphoproliferative disease (PID LPD), EBV+ acquired immunodeficiency (AID) LPD, and EBV viremia for whom there are no other approved therapeutic options, and who are not eligible to enroll in clinical studies designed to support the development and registration of tabelecleucel.
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Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
Any of the following diagnoses of EBV+ disease:
EBV+ PTLD following allogeneic hematopoietic cell transplant (HCT) or solid organ transplant (SOT) and who cannot be treated under protocols designed to support drug development or registration
Persistent EBV viremia as evidenced by 2 serial serum EBV deoxyribonucleic acid (DNA) assays showing detectable EBV viremia at least 4 weeks apart, and known or suspected immunodeficiency, or EBV viremia with a history of prior EBV+ malignancy
EBV+ LPD that has developed in the setting of an AID
EBV+ LPD that has developed in the setting of a known or suspected PID
Evidence of EBV positivity as follows:
Biopsy showing EBV+ disease, OR
A combination of circulating EBV DNA AND radiographic appearance consistent with an EBV+ malignancy, if biopsy is not clinically feasible, OR
For EBV viremia only, fulfillment of inclusion criteria 1b
Relapsed or refractory disease, which is defined as:
In patients with PTLD following HCT: relapsed after or refractory to reduction of immunosuppression and an anti-CD20 monoclonal antibody (unless investigator determines tumor to be CD20-) as a single agent or in combination with chemotherapy. Patients with CD20- disease must have failed chemotherapy alone.
In patients with PTLD following SOT: relapse after or refractory to reduction of immunosuppression, an anti-CD20 monoclonal antibody (unless investigator determines tumor to be CD20-) plus anthracycline based chemotherapy. Patients with CD20- disease must have failed chemotherapy alone.
In patients with EBV viremia: Viremia that persists or recurs despite therapy with an anti-CD20 monoclonal antibody
In patients with PID LPD: relapsed after or refractory to an anti-CD20 monoclonal antibody (unless investigator determines tumor to be CD20-) as a single agent or in combination with a chemotherapy
In patients with AID LPD: have failed at least two prior lines of therapy, one of which is an anti-CD20 monoclonal antibody (unless investigator determines tumor to be CD20-) plus anthracycline based chemotherapy (Note: Remission consolidation with an autologous or allogeneic stem cell transplant will not be considered as a separate line of therapy).
Not eligible for any other studies supporting development of tabelecleucel
For patients with PTLD in the allogeneic HCT setting, the underlying disease for which allogeneic HCT was performed is in morphologic remission
Adequate organ function per the following:
Absolute neutrophil count ≥ 500/μL, with or without cytokine support
Platelet count ≥ 20,000/μL, with or without transfusion support
Patient or patient's representative is willing and able to provide written informed consent
Burkitt's lymphoma, classical Hodgkin's lymphoma, plasmablastic lymphoma, or any T-cell lymphoma
Any investigational therapy received within 4 weeks prior to Cycle 1 Day 1, except for therapy that is outside of 5 half-lives from the most recent dose to Cycle 1 Day 1
Ongoing need for methotrexate or extracorporeal photopheresis; steroid doses > 1 mg/kg/day, as prednisone equivalent
Need for vasopressor or ventilatory support, unless deemed to be caused by the EBV-driven process that tabelecleucel is intended to treat
Antithymocyte globulin, alemtuzumab, or similar anti-T-cell antibody therapy, or T-cell immunotherapy (donor lymphocyte infusion, other CTLs) ≤ 4 weeks prior to Cycle 1 Day 1
Female of childbearing potential or male with a female partner of childbearing potential unwilling to use a highly effective method of contraception, except when, in the opinion of the treating physician in consultation with the medical monitor, the risk/benefit of tabelecleucel therapy favors proceeding