A Phase 3 Study of Tabelecleucel for Participants With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure With Rituximab or Rituximab and Chemotherapy

NCT ID: NCT03394365

Last Updated: 2026-01-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

115 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-29

Study Completion Date

2030-08-31

Brief Summary

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The purpose of this study is to determine the clinical benefit and characterize the safety profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in the setting of (1) solid organ transplant (SOT) after failure of rituximab (SOT-R) and rituximab plus chemotherapy (SOT-R+C) or (2) allogeneic hematopoietic cell transplant (HCT) after failure of rituximab.

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Detailed Description

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This is a multicenter, open-label, phase 3 study to assess the efficacy and safety of tabelecleucel for the treatment of EBV+ PTLD in the setting of SOT-R and SOT-R+C (Cohort \[C\]-SOT) or HCT after failure of rituximab (C-HCT).

SOT-R further included participants:

1. who did not receive chemotherapy and did not have a documented medical reason not to receive chemotherapy (SOT-Ro) or
2. who were considered chemotherapy ineligible/inappropriate (SOT-R-Ci)

Combined population (SOT-R-Ci, SOT-R+C, and HCT) and (SOT-R-Ci and SOT-R+C) who received commercial product, or a product manufactured using a comparable process version (PV) were also used for analysis of outcomes.

Enrollment will be preceded by confirmation of availability of partially human leukocyte antigen (HLA) matched and restricted tabelecleucel for the participant.

Study procedures and product administration will be the same for each cohort. Tabelecleucel will be administered in cycles lasting 5 weeks (35 days). During each cycle, participants will receive intravenous tabelecleucel at a dose of 2 × 10\^6 cells/kg on Days 1, 8, and 15, followed by observation through Day 35. Treatment will continue until maximal response, unacceptable toxicity, initiation of non protocol therapy, or failure of tabelecleucel with up to 2 different HLA restrictions (C-SOT) or up to 4 different HLA restrictions (C-HCT). The study includes a total of 5 years of follow-up for disease and survival status for participants enrolled before or after 09 October 2023 to reach the initial sample size of 33 participants in both cohorts. For all other participants enrolled after 09 October 2023 and after the initial sample of 33 participants in both cohorts has been reached in both cohorts, the follow-up will be every 3 months, up to 12 months, as assessed on anniversary of Cycle 1 Day 1. For responders, the follow-up will be 12 months from the date of initial response.

Conditions

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Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD) Solid Organ Transplant Complications Lymphoproliferative Disorders Allogeneic Hematopoietic Cell Transplant Stem Cell Transplant Complications

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort SOT-R (C-SOT-R)

Participants with EBV+ PTLD following SOT that has failed rituximab will receive IV tabelecleucel.

Group Type EXPERIMENTAL

tabelecleucel

Intervention Type BIOLOGICAL

Tabelecleucel is being investigated as an off-the-shelf, allogeneic T-cell immunotherapy for the treatment of EBV+ malignancies and diseases.

Cohort SOT-R+C (C-SOT-R+C)

Participants with EBV+ PTLD following SOT that has failed both rituximab and chemotherapy will receive IV tabelecleucel.

Group Type EXPERIMENTAL

tabelecleucel

Intervention Type BIOLOGICAL

Tabelecleucel is being investigated as an off-the-shelf, allogeneic T-cell immunotherapy for the treatment of EBV+ malignancies and diseases.

Cohort HCT (C-HCT)

Participants with EBV+ PTLD following HCT that has failed rituximab containing regimen will receive IV tabelecleucel.

Group Type EXPERIMENTAL

tabelecleucel

Intervention Type BIOLOGICAL

Tabelecleucel is being investigated as an off-the-shelf, allogeneic T-cell immunotherapy for the treatment of EBV+ malignancies and diseases.

Interventions

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tabelecleucel

Tabelecleucel is being investigated as an off-the-shelf, allogeneic T-cell immunotherapy for the treatment of EBV+ malignancies and diseases.

Intervention Type BIOLOGICAL

Other Intervention Names

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tab-cel® ATA129 EBV-CTL Ebvallo®

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pierre Fabre Medicament

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Anke Friedetzky

Role: STUDY_DIRECTOR

Pierre Fabre Laboratories

Locations

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City of Hope (Adults and Pediatrics)

Duarte, California, United States

Site Status COMPLETED

University of California San Diego Moores Cancer Center (Adults only)

La Jolla, California, United States

Site Status COMPLETED

Loma Linda University Medical Center (Adults only)

Loma Linda, California, United States

Site Status RECRUITING

Children's Hospital Los Angeles, Div. of Research Immunology/BMT (Adults and Pediatrics)

Los Angeles, California, United States

Site Status RECRUITING

UCLA Medical Center (Adults and Pediatrics)

Los Angeles, California, United States

Site Status COMPLETED

University of California Davis Comprehensive Cancer Center (Adults only)

Sacramento, California, United States

Site Status COMPLETED

Yale University (Adults and Pediatrics)

New Haven, Connecticut, United States

Site Status COMPLETED

MedStar Georgetown University Hospital (Adults and Pediatrics)

Washington D.C., District of Columbia, United States

Site Status RECRUITING

University of Florida (Adults and Pediatrics)

Gainesville, Florida, United States

Site Status COMPLETED

University of Miami/Jackson Memorial Hospital (Adults only)

Miami, Florida, United States

Site Status RECRUITING

Winship Cancer Institute (Adults only)

Atlanta, Georgia, United States

Site Status COMPLETED

Arthur M. Blank Hospital (Pediatrics)

Atlanta, Georgia, United States

Site Status RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago (Adults and Pediatrics)

Chicago, Illinois, United States

Site Status RECRUITING

University of Chicago Medical Center - Duchossois Center for Advanced Medicine (Adults only)

Chicago, Illinois, United States

Site Status COMPLETED

Loyola University Medical Center (Adults and Pediatrics)

Maywood, Illinois, United States

Site Status RECRUITING

University of Maryland School of Medicine (Adults only)

Baltimore, Maryland, United States

Site Status COMPLETED

Dana Farber Cancer Institute, Brigham and Women's Hospital (Adults and Pediatrics)

Boston, Massachusetts, United States

Site Status RECRUITING

Washington University School of Medicine (Adults only)

St Louis, Missouri, United States

Site Status RECRUITING

Weill Cornell Medicine (Adults only)

New York, New York, United States

Site Status COMPLETED

Columbia University Medical Center (Adults and Pediatrics)

New York, New York, United States

Site Status COMPLETED

Memorial Sloan Kettering Cancer Center (Adults and Pediatrics)

New York, New York, United States

Site Status COMPLETED

Montefiore Medical Center (Adults only)

The Bronx, New York, United States

Site Status COMPLETED

Montefiore Medical Center (Pediatrics only)

The Bronx, New York, United States

Site Status COMPLETED

University of North Carolina at Chapel Hill, Lineberger Comprehensive Cancer Center (Adults and Pediatrics)

Chapel Hill, North Carolina, United States

Site Status COMPLETED

Carolinas Medical Center/Levine Children's Hospital (Adults and Pediatrics)

Charlotte, North Carolina, United States

Site Status RECRUITING

Duke Cancer Institute (Adults and Pediatrics)

Durham, North Carolina, United States

Site Status RECRUITING

Cleveland Clinic Foundation (Adults and Pediatrics)

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital (Pediatrics only)

Columbus, Ohio, United States

Site Status COMPLETED

Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (Adults and Pediatrics)

Columbus, Ohio, United States

Site Status RECRUITING

Oregon Health and Science University Physicians Pavilion (Adults and Pediatrics)

Portland, Oregon, United States

Site Status RECRUITING

Hospital of the University of Pennsylvania (Adults only)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

The Children's Hospital of Philadelphia Oncology Division, Blood & Marrow Transplant Section (Pediatrics)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

University of Pittsburgh Medical Center (Adults only)

Pittsburgh, Pennsylvania, United States

Site Status COMPLETED

Medical University of South Carolina (Adults and Pediatrics)

Charleston, South Carolina, United States

Site Status ACTIVE_NOT_RECRUITING

Saint Jude Children's Research Hospital (Pediatrics only)

Memphis, Tennessee, United States

Site Status COMPLETED

Vanderbilt University Medical Center Henry-Joyce Cancer Clinic (Adults and Pediatrics)

Nashville, Tennessee, United States

Site Status RECRUITING

Baylor Scott and White Research Institute (Adults only)

Dallas, Texas, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center - Children's Medical Center (Pediatrics only)

Dallas, Texas, United States

Site Status RECRUITING

MD Anderson Cancer Center (Pediatrics and Adult)

Houston, Texas, United States

Site Status RECRUITING

Froedtert and Medical College of Wisconsin - Clinical Cancer Center (Adults only)

Milwaukee, Wisconsin, United States

Site Status COMPLETED

The Children's Hospital at Westmead (Pediatrics only)

Westmead, New South Wales, Australia

Site Status RECRUITING

Westmead Hospital (Adults only)

Westmead, New South Wales, Australia

Site Status RECRUITING

The Prince Charles Hospital (Adults only)

Chermside, Queensland, Australia

Site Status RECRUITING

Royal Adelaide Hospital (Adults only)

Adelaide, South Australia, Australia

Site Status RECRUITING

The Royal Children's Hospital Melbourne (Pediatrics only)

Melbourne, Victoria, Australia

Site Status RECRUITING

Fiona Stanley Hospital (Adults only)

Murdoch, Western Australia, Australia

Site Status RECRUITING

Medizinische Universitat Wien (Adults only)

Vienna, Austria, Austria

Site Status ACTIVE_NOT_RECRUITING

Centre Hospitalier Universitaire de Liège Site Sart Tilman (Adults and Pediatrics)

Liège, Brussels Capital, Belgium

Site Status COMPLETED

Universitair Ziekenhuis Leuven (Adults and Pediatrics)

Leuven, Flemish Brabant, Belgium

Site Status ACTIVE_NOT_RECRUITING

Alberta Children's Hospital (Adults and Pediatrics)

Calgary, Alberta, Canada

Site Status RECRUITING

Sick Kids (Pediatrics only)

Toronto, Ontario, Canada

Site Status RECRUITING

Princess Margaret Cancer Centre (Adults only)

Toronto, Ontario, Canada

Site Status RECRUITING

Centre Hospitalier Universitaire de Bordeaux (Adults only)

Pessac, Aquitaine, France

Site Status COMPLETED

Centre Hospitalier Régional Universitaire de Lille (Adults and Pediatrics)

Lille, Hauts-de-France, France

Site Status COMPLETED

Hôpital Necker-Enfants Malades (Pediatrics only)

Paris, Île-de-France Region, France

Site Status COMPLETED

Hôpital Saint Antoine (Adults only)

Paris, Île-de-France Region, France

Site Status COMPLETED

Hôpital Universitaire Pitié Salpêtrière (Adults only)

Paris, Île-de-France Region, France

Site Status COMPLETED

Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda (Adults only)

Milan, Milano, Italy

Site Status ACTIVE_NOT_RECRUITING

Fondazione IRCCS Policlinico San Matteo (Adults and Pediatrics)

Pavia, Pavia, Italy

Site Status COMPLETED

Fondazione Policlinico Universitario Agostino Gemelli (Adults only)

Roma, Roma, Italy

Site Status COMPLETED

Ospedale Pediatrico Bambino Gesu (Pediatrics only)

Roma, Roma, Italy

Site Status COMPLETED

Azienda Ospedaliera - Universitaria Città della Salute e della Scienza di Torino (Adults only)

Torino, Torino, Italy

Site Status COMPLETED

Hospital Duran i Reynals (Adults and Pediatrics)

Badalona, BARCELONA, Spain

Site Status RECRUITING

Hospital Universitari Vall d'Hebrón - Institut de Recerca (Adults and Pediatrics)

Barcelona, Barcelona, Spain

Site Status SUSPENDED

Hospital Universitario Marqués de Valdecilla (Adults and Pediatrics)

Santander, Cantabria, Spain

Site Status RECRUITING

Hospital General Universitario Gregorio Marañón (Adults and Pediatrics)

Madrid, Madrid, Spain

Site Status RECRUITING

University Hospital Virgen del Rocio (Adults and Pediatrics)

Seville, Spain, Spain

Site Status RECRUITING

Hospital Universitario La Fe (Adults and Pediatrics)

Valencia, Valencia, Spain

Site Status RECRUITING

University Hospitals Birmingham NHS Foundation Trust (Adults only)

Birmingham, England, United Kingdom

Site Status RECRUITING

King's College Hospital NHS Foundation Trust (Adults only)

London, England, United Kingdom

Site Status COMPLETED

Imperial College Healthcare NHS Trust (Adults only)

London, England, United Kingdom

Site Status RECRUITING

Countries

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United States Australia Austria Belgium Canada France Italy Spain United Kingdom

Central Contacts

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Claire Fabre

Role: CONTACT

6 42 04 84 76 ext. +33

Marie Bosredon

Role: CONTACT

7 84 14 59 82 ext. +33

Facility Contacts

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Sajad Khazal, MD

Role: primary

1 (909) 558-4910

Amber Medina

Role: primary

323-361-5654

Neena Kapoor, MD

Role: backup

323-361-2434

Udeme Ekong, MD

Role: primary

202-444-3514

Amer Beitinjaneh, MD, MPH, MSc

Role: primary

1 (305) 243-9848

Suhag Parikh, MD

Role: primary

1 (404) 727-8930

Sonali Chaudhury, MD

Role: primary

312-227-4090

Patrick Hagen, MD

Role: primary

1 (708) 327-2241

Sarah Nikiforow, MD

Role: primary

617-632-6640

Armin Ghobadi, MD

Role: primary

(314) 747-8439

Michael Kent, MD

Role: primary

704-381-9900

Matthew McKinney, MD

Role: primary

1 (919) 684-8111

Rabi Hanna, MD

Role: primary

1 (216) 444-0663

Robert Baiocchi, MD, PhD

Role: primary

(614) 293-3196

Andy Chen, MD

Role: primary

1 (503) 494-5058

Sunita Nasta, MD

Role: primary

215-662-6933

Caitlin Elgarten, MD

Role: primary

1 267-425-7964

Bhagirathbhai Dholaria, MD

Role: primary

1 (615) 875-3112

Luis Pineiro, MD

Role: primary

214-370-1000

Tamra Slone, MD

Role: primary

1 (214) 648-3150

Priti Tewari, MD

Role: primary

1 713-632-5087

Caroline Bateman, MD

Role: primary

2 9845 2185

Shyam Panicker, MD, MBBS, MRCP, FRACP, FRCPA

Role: primary

2 9845 6352

Chandima Divithotawela, MBBS, FRACP

Role: primary

7 3139 4000

Devendra Hiwase, MD

Role: primary

870740000

Theresa Cole, BM, MRCPCH, FRACP

Role: primary

0393455522

Duncan Purtill, MD, MBBS, FRACP, FRCPA

Role: primary

(8) 6152 3788

Victor Lewis, MD

Role: primary

(403) 955-7203

Joerg Krueger, MD

Role: primary

(416) 813-7654

Igor Novitzky Basso, MD

Role: primary

(416) 315-1147

Eva González-Barca, MD

Role: primary

932274778

Lucrecia Yanez, MD

Role: primary

942202573

Rebeca Bailen, MD

Role: primary

91 426 98 26

Jose Perez-Simon, MD

Role: primary

955 013260

Juan Montoro, MD

Role: primary

961 245 876

Sridhar Chaganti, MD

Role: primary

1214242000

Eduardo Olavarria, MD

Role: primary

208 383 2134

References

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Mahadeo KM, Baiocchi R, Beitinjaneh A, Chaganti S, Choquet S, Dierickx D, Dinavahi R, Duan X, Gamelin L, Ghobadi A, Guzman-Becerra N, Joshi M, Mehta A, Navarro WH, Nikiforow S, O'Reilly RJ, Reshef R, Ruiz F, Spindler T, Prockop S. Tabelecleucel for allogeneic haematopoietic stem-cell or solid organ transplant recipients with Epstein-Barr virus-positive post-transplant lymphoproliferative disease after failure of rituximab or rituximab and chemotherapy (ALLELE): a phase 3, multicentre, open-label trial. Lancet Oncol. 2024 Mar;25(3):376-387. doi: 10.1016/S1470-2045(23)00649-6. Epub 2024 Jan 31.

Reference Type DERIVED
PMID: 38309282 (View on PubMed)

Other Identifiers

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2024-516622-57-00

Identifier Type: CTIS

Identifier Source: secondary_id

ATA129-EBV-302/F60085DL302

Identifier Type: -

Identifier Source: org_study_id

NCT03392142

Identifier Type: -

Identifier Source: nct_alias

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