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Treatment for Epstein-Barr Virus-Associated Lymphoproliferative Disease After Transplant

Multicenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy

Phase 3 Interventional Pierre Fabre Medicament · NCT03394365

This study is testing a new treatment called tabelecleucel for patients with a specific type of cancer related to Epstein-Barr virus who haven't improved with other therapies after receiving a transplant.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment115 (estimated)
SexAll
SponsorPierre Fabre Medicament Industry-sponsored
Drugs / interventionsrituximab, ipilimumab, pembrolizumab, nivolumab, chimeric antigen receptor, chemotherapy, methotrexate, prednisone
Locations71 sites (Duarte, California and 70 other locations)
Trial IDNCT03394365 on ClinicalTrials.gov

What this trial studies

This phase 3 study evaluates the efficacy and safety of tabelecleucel for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) who have not responded to rituximab or chemotherapy. It involves participants who have undergone solid organ transplants or allogeneic hematopoietic cell transplants. The treatment consists of administering tabelecleucel in cycles over a period of 5 weeks, with follow-up for up to 5 years to assess disease response and survival outcomes.

Who should consider this trial

Good fit: Ideal candidates include individuals who have undergone solid organ or allogeneic hematopoietic cell transplants and have a confirmed diagnosis of EBV+ PTLD after failing prior treatments.

Not a fit: Patients who have not undergone a transplant or those with other types of lymphoproliferative disorders unrelated to EBV may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with EBV+ PTLD who have limited treatment alternatives.

How similar studies have performed: While there have been studies on treatments for EBV+ PTLD, the specific use of tabelecleucel in this context is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Prior SOT of kidney, liver, heart, lung, pancreas, small bowel, or any combination of these (C-SOT); or prior allogeneic HCT (C-HCT).
2. A diagnosis of locally assessed, biopsy-proven EBV+ PTLD.
3. Availability of appropriate partially HLA-matched and restricted tabelecleucel has been confirmed by the sponsor.
4. Measurable, 18F-deoxyglucose (FDG)-avid (Deauville score ≥ 3) systemic disease using Lugano Classification response criteria by positron emission tomography (PET)-diagnostic computed tomography (CT), except when contraindicated or mandated by local practice, then magnetic resonance imaging (MRI) may be used. For participants with treated central nervous system (CNS) disease, a head CT and/or brain/spinal MRI as clinically appropriate will be required to follow CNS disease response per Lugano Classification response criteria.
5. Treatment failure of rituximab or interchangeable commercially available biosimilar monotherapy (C-SOT-R or C-HCT) or rituximab plus any concurrent or sequentially administered chemotherapy regimen (C-SOT-R+C) for treatment of PTLD.
6. Males and females of any age.
7. Eastern Cooperative Oncology Group performance status ≤ 3 for participants aged ≥ 16 years; Lansky score ≥ 20 for participants \< 16 years.
8. For C-HCT only: If allogeneic HCT was performed as treatment for an acute lymphoid or myeloid malignancy, the underlying primary disease for which the participant underwent transplant must be in morphologic remission.
9. Adequate organ function.

   1. Absolute neutrophil count ≥ 1000/μL, (C-SOT) or ≥ 500/μL (C-HCT), with or without cytokine support.
   2. Platelet count ≥ 50,000/μL, with or without transfusion or cytokine support. For C-HCT, platelet count \< 50,000/μL but ≥ 20,000/μL, with or without transfusion support, is permissible if the participant has not had grade ≥ 2 bleeding in the prior 4 weeks (where grading of the bleeding is determined per the National Cancer Institute's Common Terminology Criteria for Adverse Events \[CTCAE\], version 5.0).
   3. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and total bilirubin each \< 5 × the upper limit of normal; however, ALT, AST, and total bilirubin each ≤ 10 × upper limit of normal is acceptable if the elevation is considered by the investigator to be due to EBV and/or PTLD involvement of the liver as long as there is no known evidence of significant liver dysfunction.
10. Participant or participant's representative is willing and able to provide written informed consent.

Exclusion Criteria:

1. Currently active Burkitt, T-cell, NK/T-cell lymphoma/LPD, Hodgkin, plasmablastic, transformed lymphoma, active hemophagocytic lymphohistiocytosis, or other malignancies requiring systemic therapy.
2. Daily steroids of \> 0.5 mg/kg prednisone or glucocorticoid equivalent, ongoing methotrexate, or extracorporeal photopheresis.
3. Untreated CNS PTLD or CNS PTLD for which the participant is actively receiving CNS-directed chemotherapy (systemic or intrathecal) or radiotherapy at enrollment. NOTE: Participants with previously treated CNS PTLD may enroll if CNS-directed therapy is complete.
4. Suspected or confirmed grade ≥ 2 graft-versus-host disease (GvHD) per the Center for International Blood and Marrow Transplant Research consensus grading system at enrollment.
5. Ongoing or recent use of a checkpoint inhibitor agent (eg, ipilimumab, pembrolizumab, nivolumab) within 3 drug half-lives from the most recent dose to enrollment.
6. For C-HCT: active adenovirus viremia.
7. Need for vasopressor or ventilatory support.
8. Antithymocyte globulin or similar anti-T cell antibody therapy ≤ 4 weeks prior to enrollment.
9. Treatment with Epstein-Barr virus cytotoxic T lymphocytes or chimeric antigen receptor T cells directed against B cells within 8 weeks of enrollment (C-SOT or C-HCT), or unselected donor lymphocyte infusion within 8 weeks of enrollment (C-HCT only).
10. Female who is breastfeeding or pregnant or female of childbearing potential or male with a female partner of childbearing potential unwilling to use a highly effective method of contraception.
11. Inability to comply with study-related procedures.
12. Any medical condition or organ system dysfunction that in the investigator';s opinion, could compromise the participant's safety or ability to complete the study.

Where this trial is running

Duarte, California and 70 other locations

+21 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative DiseaseSolid Organ Transplant ComplicationsLymphoproliferative DisordersAllogeneic Hematopoietic Cell TransplantStem Cell Transplant ComplicationsEpstein-Barr Virus-associated Lymphoproliferative DiseaseEpstein-Barr VirusCytotoxic T lymphocyte
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.