Study of Adverse Events and Changes in Disease Status in Pediatric Participants (and Young Adults Aged 18–25) With Relapsed/Refractory Aggressive Mature B-Cell Neoplasms Receiving Subcutaneous (SC) Injections of Epcoritamab

Diffuse large B-cell lymphoma (DLBCL) and Burkitt lymphoma are rapidly progressing forms of mature non-Hodgkin lymphoma and affect B lymphocytes, a subtype of white blood cells that are part of the body’s immune system. These lymphomas can also affect children and young adults, who are the target population of this study. In these diseases, rapid enlargement of the lymph nodes and spread to other organs can be observed. Other symptoms include, among others, fever, night sweats, and weight loss. The exact causes are unknown, but genetic mutations and infections such as the Epstein-Barr virus may play a role. Patients with newly diagnosed DLBCL are typically treated with a combination of chemotherapy and immunotherapy, such as R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone). Patients with Burkitt lymphoma usually receive combination chemotherapy according to treatment guidelines for acute lymphoblastic leukemia. In 90% of cases, this treatment results in a complete cure. However, if the disease cannot be controlled with this therapy (refractory) or if the disease returns (relapse), the treatment is switched, for example, to CAR-T cells or a stem cell transplant.

The goal of this Phase 1 study is to test the efficacy and safety of a new drug in this setting: Epcoritamab. Epcoritamab is a special antibody that binds to two different surface proteins (bispecific), thereby helping the immune system to recognize and destroy cancer cells. Among other indications, the drug is already approved for the treatment of relapsed or refractory DLBCL in adults. All participants will receive the same treatment as part of the study, and epcoritamab will be administered subcutaneously (injected under the skin) in 28-day cycles. The study is open-label, meaning that both the medical staff and the patient know which drug is being administered. Treatments and follow-up visits as part of the study will continue for at least 3 years, during which the efficacy of the therapy, the occurrence of treatment-related side effects, and the concentration of the active ingredient in the body will be evaluated. The study will be discontinued if the disease progresses or if significant side effects occur.

Patients between the ages of 1 and 25 with DLBCL, Burkitt lymphoma, or Burkitt-like lymphoma/leukemia are eligible to participate in this study. The disease must have been previously treated and must either be uncontrollable (refractory) or show signs of recurrence.