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

NCT04554914 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ATA129-EBV-205/F60085DL2052024-516623-14-00
• Study Type: interventional
• Target: 190
• Locations: 7 countries
• Sites: 40

Brief Summary

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

Conditions

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

Keywords

Allogeneic, Off-The-Shelf T-cell Immunotherapy; Epstein-Barr Virus (EBV); Epstein-Barr Virus-specific Cytotoxic T lymphocyte (EBV-CTL); Solid Organ Transplant (SOT); Hematopoietic Cell Transplant (HCT); EBVision

Outcome Measures

Primary Outcomes (1)

• Objective response rate (ORR)

  Up to 2 years

Secondary Outcomes (8)

• Overall survival (OS)

  Up to 2 years

• Duration of response (DOR)

  Up to 2 years

• Progression-free survival (PFS)

  Up to 2 years

• For EBV+ PID LPD cohort: Number of participants who reach definitive therapy (ie, allogeneic HCT) for the underlying disease

  Up to 2 years

• For EBV+ PID LPD cohort: Time to definitive therapy

  Up to 2 years

Study Arms (5)

EBV+ PID LPD

EXPERIMENTAL

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

Biological: Tabelecleucel

EBV+ AID LPD

EXPERIMENTAL

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

Biological: Tabelecleucel

EBV+ CNS PTLD

EXPERIMENTAL

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

Biological: Tabelecleucel

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

EXPERIMENTAL

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

Biological: Tabelecleucel

EBV+ sarcoma, including LMS, or smooth muscle tumors

EXPERIMENTAL

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.

Biological: Tabelecleucel

Interventions

Tabelecleucel BIOLOGICAL

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

Also known as: ATA129, EBV-CTLs

EBV+ 1L PTLD (inappropriate for first-line therapy or CD20-negative); EBV+ AID LPD; EBV+ CNS PTLD; EBV+ PID LPD; EBV+ sarcoma, including LMS, or smooth muscle tumors

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Pierre Fabre Medicament: lead

Study Sites (40)

University of California Los Angeles (UCLA) (Adults and Pediatrics)

Los Angeles, California, 90095, United States

Location

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

Orange, California, 92868, United States

Location

Lucile Packard Children's Hospital Stanford (Pediatrics only)

Palo Alto, California, 94304, United States

Location

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

Sacramento, California, 95817, United States

Location

Sylvester Comprehensive Cancer Center/ University of Miami

Miami, Florida, 33136, United States

Location

Moffit Cancer Center (Adults only)

Tampa, Florida, 33612, United States

Location

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

Atlanta, Georgia, 30322, United States

Location

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

Atlanta, Georgia, 30322, United States

Location

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

Chicago, Illinois, 60611, United States

Location

University of Maryland Medical Center (Adults only)

Baltimore, Maryland, 21201, United States

Location

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

Boston, Massachusetts, 02215, United States

Location

University of Michigan Rogel Cancer Center (Adults and Pediatrics)

Ann Arbor, Michigan, 48109, United States

Location

University of Minnesota (Adults only)

Minneapolis, Minnesota, 55455, United States

Location

Washington University in St. Louis (Adults only)

St Louis, Missouri, 63108, United States

Location

Columbia University Irving Medical Center (Adults only)

New York, New York, 10032, United States

Location

Memorial Sloan-Kettering Cancer Center (Adults and Pediatrics)

New York, New York, 10065, United States

Location

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

The Bronx, New York, 10467, United States

Location

Cleveland Clinic Taussig Cancer Center (Adults and Pediatrics)

Cleveland, Ohio, 44195, United States

Location

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

Columbus, Ohio, 43210, United States

Location

Oregon Health and Science University (Adults and Pediatrics)

Portland, Oregon, 97239, United States

Location

Medical University of South Carolina (Adults and Pediatrics)

Charleston, South Carolina, 29425, United States

Location

University of Texas Southwestern Medical Center (Pediatrics only)

Dallas, Texas, 75390, United States

Location

MD Anderson (Adults and Pediatrics)

Houston, Texas, 77030, United States

Location

Uniklinikum Salzburg Landeskrankenhaus (Adults only)

Salzburg, State of Salzburg, 5020, Austria

Location

Medizinische Universität Wien (Adults only)

Vienna, State of Vienna, 1090, Austria

Location

Medizinische Universität Graz (Adults only)

Graz, Styria, 8036, Austria

Location

Hôpital Universitaire des Enfants Reine Fabiola (Pediatrics only)

Brussels, Brussles, 1020, Belgium

Location

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

Bruges, West-Vlaanderen, 8000, Belgium

Location

Algemeen Ziekenhuis Delta - Campus Rumbeke (Adults only)

Roeselare, West-Vlaanderen, 8800, Belgium

Location

Hôpital Saint-Eloi (Adults and Pediatrics)

Montpellier, Montpellier, 34295, France

Location

Hôpital Necker-Enfants Malades (Adults and Pediatrics)

Paris, Paris, 75015, France

Location

Hôpital Universitaire Pitié Salpêtrière (Adults only)

Paris, 75013, France

Location

Azienda Ospedaliero-Universitaria Pisana (Adults only)

Pisa, Pisa, 56126, Italy

Location

Ospedale Pediatrico Bambino Gesù (Adults and Pediatrics)

Roma, Roma, 00165, Italy

Location

Ospedale Infantile Regina Margherita (Pediatrics only)

Torino, Torino, 10126, Italy

Location

Hospital Universitari Vall d'Hebrón (Adults and Pediatrics)

Barcelona, Barcelona, 08035, Spain

Location

Hospital Universitario Ramón y Cajal (Adults only)

Madrid, Madrid, 28034, Spain

Location

Hospital Universitario Virgen del Rocio (Adults and Pediatrics)

Seville, Sevilla, 41013, Spain

Location

University Hospital Birmingham NHS Foundation Trust (Adults only)

Birmingham, England, B15 2TH, United Kingdom

Location

Great Ormond Street Hospital (Pediatrics only)

London, England, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Epstein-Barr Virus Infections; Immunologic Deficiency Syndromes; Lymphoproliferative Disorders; Leiomyosarcoma

Condition Hierarchy (Ancestors)

Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Immune System Diseases; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma

Study Officials

• Glen Lew

  Pierre Fabre Laboratories

  STUDY DIRECTOR

• Federica Cattaneo

  Pierre Fabre Laboratories

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2020
• First Posted: September 18, 2020
• Study Start: July 14, 2021
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): May 1, 2028
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); GB (2); IT (3); BE (3); US (23); AU (3); FR (3)