A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases

NCT ID: NCT04554914

Last Updated: 2026-01-26

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

PHASE2

Total Enrollment

190 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-14

Study Completion Date

2028-05-31

Brief Summary

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The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with EBV-associated diseases.

Detailed Description

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This is a multicenter, multicohort, open-label, single-arm, Phase 2 study to assess the efficacy and safety of tabelecleucel for the treatment of EBV-associated diseases. Participants will be enrolled in one of the following cohorts:

* EBV+ lymphoproliferative disease (LPD) in the setting of primary immunodeficiency (PID) (EBV+ PID LPD) that is relapsed and/or refractory (R/R) or newly diagnosed where standard first-line therapy is inappropriate
* EBV+ LPD in the setting of acquired (non-congenital) immunodeficiency (AID) (EBV+ AID LPD) that is R/R or newly diagnosed where standard first-line therapy is inappropriate \[cohort closed for enrollment after completion of stage1\]
* EBV+ post-transplant lymphoproliferative disease (PTLD) involving the central nervous system (CNS) (EBV+ CNS PTLD) that is R/R or newly diagnosed where standard first-line therapy is inappropriate
* EBV+ PTLD where standard first-line therapy (rituximab or chemotherapy) is inappropriate, including cluster of differentiation antigen 20 (CD20)-negative disease
* EBV+ sarcomas, including leiomyosarcoma (LMS), or smooth muscle tumors that is rapidly progressive where standard first-line therapy is inappropriate

Tabelecleucel will be administered in cycles lasting for 35 days. During each cycle, participants will receive tabelecleucel at a dose of 2 x 10\^6 cells/kg intravenously (IV) weekly for 3 weeks, followed by observation through Day 35. Treatment will continue until maximal response, disease progression, unacceptable toxicity, or initiation of nonprotocol therapy for the underlying disease. For EBV+ sarcoma cohort, treatment will continue until disease progression, unacceptable toxicity, two consecutive complete responses (CRs), or up to 12 months from the first dose. Participants who fail to respond to initial tabelecleucel treatment may continue tabelecleucel with a different human leukocyte antigen (HLA) restriction (termed a Restriction Switch), if available; administration of tabelecleucel with up to four different HLA restrictions is allowed for any participant.

After treatment is completed or discontinued, participants will complete a safety follow-up visit at 30 days after the last dose and then will enter a quarterly follow-up period. Participants without documented disease progression will be assessed every 3 months after the safety follow-up visit for continued evaluation of disease response until the end of study (EOS) visit at which occurs at 12 (± 1) months after Cycle 1 Day 1 for adults, and 24 (± 1) months after Cycle 1 Day 1 for pediatric participants. Participants with disease progression any time prior to the EOS visit will continue to be followed every 3 months for survival status until the EOS visit.

An adaptive 2-stage design will be used for each relevant cohort in this study. For each cohort, 8 evaluable participants will be enrolled in Stage 1. The decision to move to Stage 2 enrollment will be based on an interim analysis of the first 8 evaluable participants in the cohort using investigator's assessment (per defined radiologic, clinical, and/or laboratory response criteria) who receive tabelecleucel and have at least 1 valid postbaseline disease response assessment. The number of participants enrolled in Stage 2 for each cohort will depend on the number of observed responders in Stage 1. Sponsor may decide not to move forward to Stage 2 in any cohort even if the criteria to move forward for that cohort are met. The decision not to move forward may also be based on data from one or other cohorts.

Conditions

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Epstein-Barr Virus (EBV)-Associated Diseases EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD) EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD) EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD) EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD) Solid Organ Transplant Complications Lymphoproliferative Disorders Allogeneic Hematopoietic Cell Transplant Stem Cell Transplant Complications EBV+ Sarcomas Leiomyosarcoma

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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EBV+ PID LPD

Participants with R/R or newly diagnosed EBV+ PID LPD for whom standard first-line therapy is inappropriate, 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.

EBV+ AID LPD

Participants with R/R or newly diagnosed EBV+ AID LPD for whom standard first-line therapy is inappropriate, will receive IV tabelecleucel.

Cohort closed for enrollment after completion of stage1

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.

EBV+ CNS PTLD

Participants with R/R or newly diagnosed EBV+ CNS PTLD for whom standard first-line therapy is inappropriate, 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.

EBV+ 1L PTLD (inappropriate for first-line therapy or CD20-negative)

Participants with EBV+ PTLD for whom standard first-line therapy (rituximab or chemotherapy) is inappropriate, including CD20-negative disease, 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.

EBV+ sarcoma, including LMS, or smooth muscle tumors

Participants with newly diagnosed EBV+ sarcoma for whom the standard first-line therapy is inappropriate, including LMS or smooth muscle tumor, 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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ATA129 EBV-CTLs

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of EBV+ disease.
* Eastern Cooperative Oncology Group performance status ≤ 3 for participants aged ≥ 16 years; Lansky score ≥ 20 for participants from ≥ 1 year to \< 16 years.
* Adequate organ function test results, unless organ dysfunction is considered to be due to the underlying EBV-associated disease by the investigator.


* For participants with CNS PTLD:

* R/R or newly diagnosed EBV+ CNS PTLD for whom the standard. first-line therapy is inappropriate, as determined by the investigator. The CNS PTLD is histologically confirmed by at least biopsy-proven EBV+ CNS PTLD or positive CSF cytology with or without radiographically measurable intracranial disease with EBV detected in CSF.
* Participants with R/R disease must have had at least one prior line of systemic therapy and one of the following: radiographic disease progression per Lugano Classification during or after treatment or failure to achieve a CR or PR (defined by Lugano radiographic criteria) after standard first-line therapy.
* Participant may have systemic and CNS disease or CNS disease only.

Exclusion Criteria

* Currently active Burkitt, T-cell, natural killer/T-cell lymphoma/LPD, Hodgkin, plasmablastic, transformed lymphoma, active hemophagocytic lymphohistiocytosis, or other malignancies requiring systemic therapy.
* Serious known active infections, defined as ongoing uncontrolled adenovirus infection or infections requiring systemic therapy at the time of enrollment, or known history of human immunodeficiency virus (HIV) infection.
* Suspected or confirmed Grade ≥ 2 acute graft-versus-host disease (GvHD) per the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus grading system or extensive chronic GvHD per National Institutes of Health (NIH) consensus criteria at the time of the enrollment.
* Need for vasopressor or ventilatory support at the time of enrollment.
* Prior therapy (in order of increasing washout period) prior to enrollment as follows:

* Within 4 weeks or 5 half-lives (whichever is shorter) for any investigational product and/ or any chemotherapy (systemic or intrathecal), targeted small molecule therapy, or antibody/biologic therapy. Note: prior anti-CD20 antibody use is permitted within the washout period if a subsequent disease response assessment indicates disease progression.
* Within 8 weeks: prior tabelecleucel (\> 8 weeks prior to enrollment) is permitted if response was obtained or if usual protocol-directed therapeutic options were not exhausted, for cellular therapies (chimeric antigen receptor therapies directed at T-cells or T-cell subsets, donor lymphocyte infusion, other CTLs or virus-specific T-cells); and/or therapies which could impact tabelecleucel function (anti-thymocyte globulin, alemtuzumab).
* Any prior treatment with EBV-CTLs with the exception of tabelecleucel as above
* Women who are breastfeeding or pregnant.
* Unwilling to comply with protocol specified contraceptive/reproductive restrictions from enrollment through 90 days after the last treatment.
* Inability or unwillingness to comply with all study procedures.
* Ongoing need for daily steroids of \> 0.5 mg/kg prednisone or glucocorticoid equivalent, ongoing methotrexate, or extracorporeal photopheresis (for participants with CNS disease, protocol-specified dexamethasone is permitted and concludes by the time of enrollment).
* Any conditions that may put the study outcomes at undue risk (life expectancy \< 60 days or any life-threatening illness, medical condition, or organ system dysfunction).
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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University of California Los Angeles (UCLA) (Adults and Pediatrics)

Los Angeles, California, United States

Site Status COMPLETED

Children's Hospital of Orange County (Pediatrics [up to 25 years old])

Orange, California, United States

Site Status RECRUITING

Lucile Packard Children's Hospital Stanford (Pediatrics only)

Palo Alto, California, United States

Site Status RECRUITING

University of California Davis Comprehensive Cancer Center (Adults and Pediatrics)

Sacramento, California, United States

Site Status COMPLETED

Sylvester Comprehensive Cancer Center/ University of Miami

Miami, Florida, United States

Site Status RECRUITING

Moffit Cancer Center (Adults only)

Tampa, Florida, United States

Site Status COMPLETED

Children's Healthcare of Atlanta (Pediatrics only [up to 25 years old])

Atlanta, Georgia, United States

Site Status RECRUITING

Emory University/Winship Cancer Institute (Adults [>= 16 years])

Atlanta, Georgia, United States

Site Status COMPLETED

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

Chicago, Illinois, United States

Site Status RECRUITING

University of Maryland Medical Center (Adults only)

Baltimore, Maryland, United States

Site Status COMPLETED

Dana Farber Cancer Institute (DFCI) (Adults and Pediatrics)

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan Rogel Cancer Center (Adults and Pediatrics)

Ann Arbor, Michigan, United States

Site Status RECRUITING

University of Minnesota (Adults only)

Minneapolis, Minnesota, United States

Site Status RECRUITING

Washington University in St. Louis (Adults only)

St Louis, Missouri, United States

Site Status RECRUITING

Columbia University Irving Medical Center (Adults only)

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

The Children's Hospital at Montefiore (Adults and Pediatrics)

The Bronx, New York, United States

Site Status RECRUITING

Cleveland Clinic Taussig Cancer Center (Adults and Pediatrics)

Cleveland, Ohio, United States

Site Status COMPLETED

The Ohio State University - The James Cancer Hospital and Solove Research Institute (Adults only)

Columbus, Ohio, United States

Site Status RECRUITING

Oregon Health and Science University (Adults and Pediatrics)

Portland, Oregon, United States

Site Status RECRUITING

Medical University of South Carolina (Adults and Pediatrics)

Charleston, South Carolina, United States

Site Status COMPLETED

University of Texas Southwestern Medical Center (Pediatrics only)

Dallas, Texas, United States

Site Status RECRUITING

MD Anderson (Adults and Pediatrics)

Houston, Texas, United States

Site Status RECRUITING

Uniklinikum Salzburg Landeskrankenhaus (Adults only)

Salzburg, Salzburg, Austria

Site Status COMPLETED

Medizinische Universität Graz (Adults only)

Graz, Styria, Austria

Site Status COMPLETED

Medizinische Universität Wien (Adults only)

Vienna, Vienna, Austria

Site Status RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola (Pediatrics only)

Brussels, Brussles, Belgium

Site Status RECRUITING

Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan (Adults only)

Bruges, West-Vlaanderen, Belgium

Site Status RECRUITING

Algemeen Ziekenhuis Delta - Campus Rumbeke (Adults only)

Roeselare, West-Vlaanderen, Belgium

Site Status RECRUITING

Hôpital Saint-Eloi (Adults and Pediatrics)

Montpellier, Montpellier, France

Site Status RECRUITING

Hôpital Necker-Enfants Malades (Adults and Pediatrics)

Paris, Paris, France

Site Status RECRUITING

Hôpital Universitaire Pitié Salpêtrière (Adults only)

Paris, , France

Site Status RECRUITING

Azienda Ospedaliero-Universitaria Pisana (Adults only)

Pisa, Pisa, Italy

Site Status RECRUITING

Ospedale Pediatrico Bambino Gesù (Adults and Pediatrics)

Roma, Roma, Italy

Site Status RECRUITING

Ospedale Infantile Regina Margherita (Pediatrics only)

Torino, Torino, Italy

Site Status RECRUITING

Hospital Universitari Vall d'Hebrón (Adults and Pediatrics)

Barcelona, Barcelona, Spain

Site Status RECRUITING

Hospital Universitario Ramón y Cajal (Adults only)

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Universitario Virgen del Rocio (Adults and Pediatrics)

Seville, Sevilla, Spain

Site Status RECRUITING

University Hospital Birmingham NHS Foundation Trust (Adults only)

Birmingham, England, United Kingdom

Site Status RECRUITING

Great Ormond Street Hospital (Pediatrics only)

London, England, United Kingdom

Site Status RECRUITING

Countries

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United States Austria Belgium France Italy Spain United Kingdom

Central Contacts

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Claire Fabre

Role: CONTACT

6 42 04 84 76 ext. +33

Cindy Jachym

Role: CONTACT

6 22 38 92 79 ext. +33

Facility Contacts

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Lilibeth Torno, MD

Role: primary

7145094348

Lianna Marks, MD

Role: primary

(650) 497-8953

Warren Alperstein, MD

Role: primary

3052437925

Shanmuganathan Chandrakasan, MD

Role: primary

404-727-8877

Sonali Chaudhury, MD

Role: primary

(312) 227-4090

Sarah Nikiforow, MD

Role: primary

6176323477

Monalisa Ghosh, MD

Role: primary

734-232-4484

Supriya Gupta, MD

Role: primary

612-626-5906

Armin Ghobadi, MD

Role: primary

314 747 2743

David Loeb, MD

Role: primary

(718) 920-4664

Robert Baiocchi, MD

Role: primary

(614)915-2084

Andy Chen, MD

Role: primary

503 494 5058

Victor Aquino, MD

Role: primary

(214) 648-8800

Priti Tewari, MD

Role: primary

713 632 5087

Nina Worel, MD

Role: primary

01 40 400 5302

Christine Devalck, MD

Role: primary

24773113

Sylvia Snauwaert, MD

Role: primary

50452111

Dries Deeran, MD

Role: primary

51237656

Charles Herbaux, MD

Role: primary

4 67 33 67 33

Felipe Suarez, MD

Role: primary

1 44 49 52 87

Sylvain S Choquet, MD

Role: primary

1 42 16 28 26

Enrico Orciuolo, MD

Role: primary

05 0993488

Franco Locatelli, MD

Role: primary

06 68592129

Franca Fagioli, MD

Role: primary

01 13135230

Pere Barba Suñol, MD

Role: primary

934893806

Javier López Jiménez, MD

Role: primary

91-336-80-00

Jose Antonio Perez Simon, MD

Role: primary

9 55 01 31 61

Sridhar Chaganti, MD

Role: primary

012 1 371 4379

Persis Amrolia, MD

Role: primary

020 7813 8434

Other Identifiers

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2024-516623-14-00

Identifier Type: CTIS

Identifier Source: secondary_id

ATA129-EBV-205/F60085DL205

Identifier Type: -

Identifier Source: org_study_id

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