Study of the Adverse Events and Change in Disease State of Pediatric Participants (and Young Adults Between the Ages of 18-25) With Relapsed/Refractory Aggressive Mature B-cell Neoplasms Receiving Subcutaneous (SC) Injections of Epcoritamab

AbbVie (Industry)
Overall Status
Recruiting ID
Genmab (Industry)

Study Details

Study Description

Brief Summary

The most common types of mature B-cell lymphomas (MBLs) in children are Burkitt lymphoma (BL) and diffuse large B-cell lymphoma (DLBCL). Initial treatment cures 90% - 95% of children with these malignancies, leaving a very small population of relapsed/refractory disease with a poor prognosis. The purpose of this study is to assess the safety and tolerability of epcoritamab in pediatric participants with relapsed/refractory aggressive mature B-cell neoplasms and young adult participants with Burkitt's or Burkitt-like lymphoma/leukemia. Adverse events and change in disease activity will be assessed.

Epcoritamab is an investigational drug being developed for the treatment of relapsed/refractory aggressive mature B-cell neoplasms. Participants will receive subcutaneous (SC) of epcoritamab. Approximately 15 pediatric participants with a diagnosis of relapsed/refractory aggressive mature B-cell neoplasms and and young adult participants, ages of 18-25, with a diagnosis of Burkitt's or Burkitt-like lymphoma/leukemia will be enrolled at 50 sites globally.

Participants will receive subcutaneous epcoritamab in 28-day cycles. Participants will be followed for a minimum of 3 years after enrollment.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at an approved institution (hospital or clinic). The effect of the treatment will be frequently checked by medical assessments, blood tests, questionnaires and side effects.

Condition or Disease Intervention/Treatment Phase
Phase 1

Study Design

Study Type:
Anticipated Enrollment :
15 participants
Intervention Model:
Single Group Assignment
None (Open Label)
Primary Purpose:
Official Title:
A Single Arm, Open-Label, Phase 1b Trial of Epcoritamab in Pediatric Patients With Relapsed/Refractory Aggressive Mature B-cell Neoplasms
Actual Study Start Date :
Jun 7, 2022
Anticipated Primary Completion Date :
Jun 18, 2027
Anticipated Study Completion Date :
Jun 18, 2027

Arms and Interventions

Arm Intervention/Treatment
Experimental: Epcoritamab

Participants will receive subcutaneous (SC) epcoritamab in 28 day cycles.

Drug: Epcoritamab
Subcutaneous Injection (SC)
Other Names:
  • ABBV-GMAB-3013
  • Outcome Measures

    Primary Outcome Measures

    1. Number of Participants with Adverse Events (AE) [Up to Approximately 3 Years]

      An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

    2. Maximum Observed Concentration (Cmax) [Up to Approximately Week 37]

      Maximum observed concentration.

    3. Area Under the Concentration Versus Time Curve (AUC) from Time 0 to Time of Last Measurable Concentration within the Dosing Interval (AUCtau) [Up to Approximately Week 37]

      AUC from time 0 to time of last measurable concentration within the dosing interval.

    Secondary Outcome Measures

    1. Percentage of Participants who Achieve Complete Response (CR) [Up to Approximately 1 Year]

      CR is defined per the International Pediatric Non-Hodgkin Lymphoma Response Criteria as computed tomography (CT) or magnetic resonance imaging (MRI) reveals no residual disease or new lesions; Resected residual mass that is pathologically (morphologically) negative for disease (detection of disease with more sensitive techniques); bone marrow (BM) and cerebrospinal fluid (CSF) morphologically free of disease (detection of disease with more sensitive techniques).

    2. Number of Participants with Event-free survival (EFS) [Up to Approximately 3 Years]

      EFS will be defined as the number of days from screening to the date of disease progression, treatment failure, or death from any cause.

    3. Number of Participants who Achieve Overall Survival (OS) [Up to Approximately 3 Years]

      OS will be defined as the number of days from screening to the date of death from any cause.

    4. Rate of Initiation of Stem Cell Transplantation or Chimeric Antigen Receptor T-cell (CAR-T) Therapy [Up to Approximately 1 Year]

      Rate of initiation of stem cell transplantation or CAR-T therapy.

    5. Percentage of Participants Achieving Overall Response (OR) [Up to Approximately 1 Year]

      OR is assessed as the percentage of participants with an overall response.

    6. Duration of response (DOR) [Up to Approximately 1 Year]

      DOR is defined as the time between the date of first response to the date of the first documented tumor progression or death due to any cause, whichever comes first.

    7. Duration of CR (DOCR) [Up to Approximately 1 Year]

      DOCR is defined as the time between the date of first CR to the date of the first documented tumor progression or death due to any cause, whichever comes first.

    8. Percentage of Participants Achieving Immunogenicity [Up to Approximately Week 37]

      Immunogenicity is defined the percentage of participants with ADA and neutralizing anti-drug antibodies (nAb).

    Eligibility Criteria


    Ages Eligible for Study:
    1 Year to 25 Years
    Sexes Eligible for Study:
    Accepts Healthy Volunteers:
    Inclusion Criteria:
    • Participants >= 1 and < 18 years old at time of primary diagnosis with Burkitt's or Burkitt-like lymphoma/leukemia, diffuse large B-cell lymphoma (DLBCL), or other aggressive mature (CD20+) B-cell lymphomas. Participants up to 25 years of age with Burkitt's or Burkitt-like lymphoma/leukemia are also eligible.

    • Disease pathologically confirmed (tumor tissue) by local testing.

    • Relapsed or primary refractory disease meeting any of the following criteria:

    • Progressive disease at any time during second-line chemoimmunotherapy (CIT).

    • Best response of stable disease (SD) after a minimum of 2 cycles of second-line CIT.

    • Best response of partial response (PR) after a minimum of 3 cycles of second-line CIT.

    • Complete Response (CR) after a minimum of 3 cycles of second-line CIT therapy but unfit or ineligible for consolidation with cell therapy.

    • Not in CR and unable to initiate or tolerate (i.e., must discontinue) second-line CIT.

    • Have received cell therapy (allogeneic or autologous transplant or chimeric antigen receptor T-cell (CAR-T) therapy) as consolidation but have not obtained or maintained a CR.

    • Recovery from toxic effects of prior chemoimmunotherapy.

    • Performance status by Lansky (< 16 years old at evaluation) or Karnofsky (>= 16 years old at evaluation) score >= 50 or Eastern Cooperative Oncology Group (ECOG) score <= 2 .

    • Adequate bone marrow, hepatic, and renal function.

    Exclusion Criteria:
    • Known central nervous system (CNS) involvement by lymphoma at screening as confirmed by screening magnetic resonance imaging (MRI)/computed tomography (CT)/positron emission tomography (PET) brain scans (participants with evidence of CNS disease only in the cerebrospinal fluid (CSF) will be eligible).

    • Other malignancy requiring therapy.

    • Currently receiving anti-cancer therapy, including chemotherapy (excluding intrathecal therapy), radiotherapy, small molecules, monoclonal antibodies, cell therapy, or other investigational agents.

    Contacts and Locations


    Site City State Country Postal Code
    1 Lucile Packard Children's Hospitals - Stanford /ID# 240854 Palo Alto California United States 94304-5786
    2 Nicklaus Children's Hospital /ID# 241174 Miami Florida United States 33155-3009
    3 New York Medical College /ID# 239208 Valhalla New York United States 10595
    4 Levine Children's Hospital /ID# 242765 Charlotte North Carolina United States 28203-5812
    5 Cincinnati Childrens Hospital Medical Center /ID# 239823 Cincinnati Ohio United States 45229
    6 Oregon Health & Science University /ID# 240090 Portland Oregon United States 97239-3011
    7 Children's Hospital of Philadelphia - Main /ID# 239294 Philadelphia Pennsylvania United States 19104-4319
    8 St Jude Children's Research Hospital /ID# 239184 Memphis Tennessee United States 38105
    9 University of Texas Southwestern Medical Center /ID# 240892 Dallas Texas United States 75390-7208
    10 Medical College of Wisconsin - Plank Rd /ID# 239782 Milwaukee Wisconsin United States 53226-3548
    11 The Children's Hospital at Westmead /ID# 240091 Sydney New South Wales Australia 2145
    12 Royal Children's Hospital /ID# 240384 Parkville Victoria Australia 3052
    13 Perth Children's Hospital /ID# 240382 Nedlands Western Australia Australia 6009
    14 Universitair Ziekenhuis Leuven /ID# 242384 Leuven Vlaams-Brabant Belgium 3000
    15 Hospital for Sick Children /ID# 240767 Toronto Ontario Canada M5G 1X8
    16 CHU Sainte-Justine /ID# 240766 Montreal Quebec Canada H3T 1C5
    17 Fakultni Nemocnice Brno /ID# 239956 Brno Czechia 625 00
    18 CHU de Nantes, Hotel Dieu -HME /ID# 240831 Nantes Pays-de-la-Loire France 44000
    19 Institut Gustave Roussy /ID# 240966 Villejuif Cedex Val-de-Marne France 94805
    20 CHU Bordeaux - Hopital Pellegrin /ID# 240832 Bordeaux France 33000
    21 Hospices Civils de Lyon /ID# 240834 Lyon France 69003
    22 Universitaetsklinikum Erlangen /ID# 240861 Erlangen Bayern Germany 91054
    23 Universitaetsklinikum Muenster /ID# 239970 Muenster Nordrhein-Westfalen Germany 48149
    24 Universitaetsklinikum Giessen und Marburg /ID# 240787 Marburg Germany 35043
    25 Universitaetsmedizin Rostock /ID# 240891 Rostock Germany 18057
    26 The Chaim Sheba Medical Center /ID# 240670 Ramat Gan Tel-Aviv Israel 5265601
    27 Rambam Health Care Campus /ID# 240037 Haifa Israel 3109601
    28 Schneider Children's Medical Center /ID# 240171 Petah Tikva Israel 4920235
    29 IRCCS Ospedale Pediatrico Bambino Gesu /ID# 240039 Rome Lazio Italy 00165
    30 Comitato Maria Letizia Verga /ID# 245592 Monza MB Monza E Brianza Italy 20900
    31 Azienda Ospedaliero Universitaria Meyer /ID# 240049 Florence Italy 50139
    32 NHO Nagoya Medical Center /ID# 246680 Nagoya-shi Aichi Japan 460-0001
    33 Kyoto University Hospital /ID# 246907 Kyoto-shi Kyoto Japan 606-8507
    34 Osaka City General Hospital /ID# 246906 Osaka-shi Osaka Japan 534-0021
    35 National Cancer Center Hospital /ID# 246722 Chuo-ku Tokyo Japan 104-0045
    36 National Center for Child Health and Development /ID# 246658 Setagaya-ku Tokyo Japan 157-8535
    37 Seoul National University Hospital /ID# 239894 Seoul Korea, Republic of 03080
    38 Samsung Medical Center /ID# 239895 Seoul Korea, Republic of 06351
    39 Prinses Maxima Centrum /ID# 239815 Utrecht Netherlands 3584 CS
    40 Hospital Universitario Vall d'Hebron /ID# 240715 Barcelona Spain 08035
    41 Hospital Infantil Universitario Nino Jesus /ID# 240717 Madrid Spain 28009
    42 Hospital Universitario La Paz /ID# 240716 Madrid Spain 28046
    43 National Taiwan University Hospital /ID# 242890 Taipei City Taiwan 100
    44 Koc Universitesi Hastanesi Translasyonel Tip Arastirma Merkezi /ID# 240026 Istanbul Turkey 34010
    45 Dokuz Eylul University Medical Faculty /ID# 239954 Izmir Turkey 35340

    Sponsors and Collaborators

    • AbbVie
    • Genmab


    • Study Director: ABBVIE INC., AbbVie

    Study Documents (Full-Text)

    None provided.

    More Information


    None provided.
    Responsible Party:
    AbbVie Identifier:
    Other Study ID Numbers:
    • M20-429
    • 2021-004555-16
    First Posted:
    Jan 25, 2022
    Last Update Posted:
    Aug 16, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Plan to Share IPD:
    Studies a U.S. FDA-regulated Drug Product:
    Studies a U.S. FDA-regulated Device Product:
    Product Manufactured in and Exported from the U.S.:
    Keywords provided by AbbVie
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Aug 16, 2022