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Epcoritamab as Consolidative Therapy for Waldenstr(SqrRoot)(Delta)m Macroglobulinemia (WM) Treated With a Bruton Tyrosine Kinase Inhibitor (BTKi)

3 de novembro de 2026 atualizado por: National Heart, Lung, and Blood Institute (NHLBI)

Epcoritamab as Consolidative Therapy for Waldenstrom Macroglobulinemia (WM) Treated With a Bruton Tyrosine Kinase Inhibitor (BTKi)

Background:

Waldenstrom macroglobulinemia (WM) is a type of blood cancer. It causes the blood to get thicker, which slows circulation and makes it harder to carry oxygen and other substances around the body. WM is treated with drugs called Bruton tyrosine kinase inhibitors (BTKi). But WM never goes away completely, and sometimes it gets worse despite treatment.

Objective:

To test a new drug (epcoritamab) in people with WM.

Eligibility:

People aged 18 years or older who have WM. They must have been taking a BTKi for a year or more.

Design:

Participants will be screened. They will have blood and urine tests. They will have imaging scans and a test of their heart function. They may have a bone marrow biopsy: A large needle will be used to remove a sample of the soft tissue and fluids from inside the hip bone.

Epcoritamab is injected under the skin, normally on the lower belly or thigh. Participants will be treated in 28-day cycles. They will have 12 treatment cycles. During the first cycle, they will come to the clinic to get an injection once a week for 4 weeks. After that, they will get 1 injection on the first day of each cycle.

Blood tests and imaging scans will be repeated at study visits. A saliva sample will be taken for genetic testing. Participants will also continue their normal treatment with BTKi.

After their last treatment with the study drug, participants will have 5 follow-up visits in 6 months. Tests, including a bone marrow biopsy, may be repeated. Follow-up visits will continue every 6 months for 3 (Omega) years or more.

Visão geral do estudo

Status

Ainda não está recrutando

Intervenção / Tratamento

Descrição detalhada

Study Description:

This is a phase 2 study of adding epcoritamab as consolidative therapy for subjects with Waldenstrom macroglobulinemia (WM) receiving treatment with a Bruton tyrosine kinase inhibitor (BTKi), zanubrutinib or ibrutinib. Subjects will be treated with epcoritamab for twelve 28-day cycles. For cycle 1, patients will receive epcoritamab subcutaneously (SC) with step-up dosing (SUD; 0.16 mg on C1D1, 0.8 mg on C1D8, 3 mg on C1D15, 48 mg on C1D22). The first 3 subjects will start treatment with epcoritamab >=4 weeks apart and be hospitalized for 24 hours after the first full dose of 48 mg for observation. In the absence of grade >=3 cytokine release syndrome (CRS), subsequent subjects will be treated on an outpatient basis. Epcoritamab 48 mg SC will be administered every 4 weeks from cycles 2 to 12.

Subjects achieving complete remission (CR) or very good partial remission (VGPR) by serum IgM after 12 cycles of epcoritamab will be evaluated by bone marrow biopsy and aspirate 2 months after the last dose of epcoritamab, and elect to discontinue BTKi therapy if CR/VGPR is confirmed. For subjects who do not attain CR/VGPR, BTKi will continue as per standard of care.

Objectives:

Primary objective:

-Evaluate the efficacy of epcoritamab as consolidative therapy in WM treated with a BTKi

Secondary objectives:

  • Evaluate the safety of epcoritamab as consolidative therapy in WM treated with a BTKi
  • Evaluate other parameters of efficacy of epcoritamab as consolidative therapy in WM treated with a BTKi

Endpoints:

Primary Endpoint:

-Rate of CR/VGPR after 12 cycles of epcoritamab in WM treated with a BTKi

Secondary Endpoint:

  • Rate of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and IgM flare
  • Incidence and severity of treatment-emergent adverse events (TEAEs)
  • Progression-free survival (PFS)
  • Duration of response after BTKi discontinuation
  • Overall survival (OS)

Tipo de estudo

Intervencional

Inscrição (Estimado)

24

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Estude backup de contato

Locais de estudo

    • Maryland
      • Bethesda, Maryland, Estados Unidos, 20892
        • National Institutes of Health Clinical Center
        • Contato:
          • NIH Clinical Center Office of Patient Recruitment (OPR)
          • Número de telefone: TTY dial 711 800-411-1222
          • E-mail: ccopr@nih.gov

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

18 anos a 100 anos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

  • INCLUSION CRITERIA:

Patients must meet the following entry criteria to be eligible to participate in the study:

  1. Age >=18 years

  2. Ability to comprehend the investigational nature of the study and provide informed consent

  3. Diagnosis of WM according to 11th International Workshop on WM (IWWM-11) (19)

    1. Presence of an IgM monoclonal protein
    2. Bone marrow involvement by lymphoplasmacytic lymphoma
  4. Presence of MYD88 L265P mutation

  5. Currently receiving a BTKi, zanubrutinib or ibrutinib, for >=12 months

  6. Able to obtain a commercial supply of BTKi

  7. Achieved partial response, minor response, or stable disease on a BTKi per IWWM-11 response criteria (19)

  8. A female participant is eligible to participate if not pregnant or breastfeeding, and >=1 of the following conditions applies:

    Is not a woman of childbearing potential (WOCBP)

    OR

    Is a WOCBP and

    • Uses a contraceptive method
    • Has a negative highly sensitive serum pregnancy test within 7 days before the first dose of study intervention.
  9. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 within 7 days prior to the first dose of study intervention.

  10. The ability to swallow and retain oral medication.

  11. Participants who are HBsAg positive are eligible if they have received HBV antiviral therapy for >=4 weeks and have undetectable HBV viral load prior to study entry.

    Note: Participants should remain on anti-viral therapy throughout study intervention and for at least 12 months following discontinuation of epcoritamab. Hepatitis B screening tests should include HBsAg and anti-HBV.

  12. Participants who received treatment for HCV that was intended to eradicate the virus are eligible if hepatitis C RNA levels are undetectable.

  13. COVID 19 eligibility criteria:

    Subject has no known active severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection. If a subject has signs/symptoms suggestive of SARS-CoV-2 infection or have had recent known exposure to someone with SARS-CoV-2 infection, the subject must have a negative molecular (e.g., PCR) test, or 2 negative antigen test results at least 24 hours apart, to rule out SARS-CoV-2 infection.

    Subjects who do not meet SARS-CoV-2 infection eligibility criteria must be screen failed and may only rescreen after they meet the following SARS-CoV-2 infection viral clearance criteria:

    • No signs/symptoms suggestive of active SARS-CoV-2 infection
    • Negative molecular (e.g., PCR) result or 2 negative antigen test results at least 24 hours apart
  14. Adequate organ function as defined below.

**Adequate Organ Function Laboratory Values**

System

Laboratory Value

Hematological

Absolute neutrophil count (ANC)

>=1000/microL (a)

Platelets

>=75000/microL or >=50000/microLin the presence of bone marrow involvement or splenomegaly (a)

Hemoglobin

>=8 g/dL (a)

Renal

Creatinine

<=1.5 x ULN or eGFR >=30 mL/min

Hepatic

Total bilirubin

<=1.5 x ULN unless due to Gilbert's syndrome or controlled autoimmune hemolytic anemia (not requiring immunosuppressive other than <=20 mg of prednisolone daily)

AST and ALT

<=3 ULN

Coagulation

PT

aPTT

<=1.5 x ULN unless participant is receiving anticoagulant therapy

eGFR=estimated glomerular filtration rate; ALT =alanine aminotransferase; AST =aspartate aminotransferase; ULN=upper limit of normal; INR=international normalized ratio; PT=prothrombin time; aPTT=activated partial thromboplastin time

  1. Growth factor and/or transfusion support is allowed to meet this requirement at Screening only if directly attributable to WM infiltration of the bone marrow, proven by bone marrow biopsy

    EXCLUSION CRITERIA:

    An individual who meets any of the following criteria will be excluded from participation in this study:

    1. Active CNS involvement, including Bing-Neel syndrome

    2. Active HBV/HCV infection.

    3. Infection with HIV-1 or HIV-2. Exception: patients with well-controlled HIV (e.g., CD4 >350/mm^3 and undetectable viral load) are eligible.

    4. Gastrointestinal dysfunction that may affect drug absorption (e.g., gastric bypass surgery, gastrectomy).

    5. Ongoing active bacterial, viral, fungal, mycobacterial, parasitic, or other infection requiring systemic treatment (excluding prophylactic treatment) at the time of enrollment or within the previous 2 weeks prior to the first dose of epcoritamab, including COVID-19 infection. Note that a past COVID-19 infection may be a risk factor, but if resolved and the subject is vaccinated, it may be allowable to enroll the Subject.

    6. Stroke or intracranial hemorrhage within 6 months of screening

    7. Hypertensive urgency or emergency

    8. Active, clinically significant cardiovascular disease including:

    9. Uncontrolled or symptomatic arrhythmias

    10. Class 3 or 4 congestive heart failure as defined by New York Heart Association Functional Classification

      • Association and/or known decreased ejection fraction <45%
    11. Myocardial infarction, unstable angina or acute coronary syndrome within 6 months of screening.

    12. History of Mobitz II second-degree or third-degree heart block without a permanent pacemaker in place.

    13. QTcF >450 msecs based on Fridericia's formula

      Note: QTcF value may be calculated as the numerical average of up to 3 separate readings for eligibility).

    14. Known allergy/sensitivity to epcoritamab

    15. History of severe bleeding disorder defined as an ongoing congenital or acquired condition that leads to an increased likelihood of bleeding (i.e., von Willebrand's disease or hemophilia).

    16. History of a second malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years.

      Note: The time requirement does not apply to participants who underwent successful definitive resection of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ, excluding carcinoma in situ of the bladder.

    17. Currently being treated with the following drugs:

      • CYP3A strong inducers
      • CYP3A strong inhibitors

      Note: A washout period of at least 5 times the half-life after the last dose of any of the above treatments is required for a participant to be eligible for study enrollment.

    18. Has received prior systemic anti-WM therapy besides a BTKi within 3 half-lives or 4 weeks (if prior therapy was a monoclonal antibody) of start of epcoritamab.

    19. Has received prior radiotherapy within 2 weeks of start of epcoritamab or radiation-related toxicities requiring corticosteroids.

      Note: Two weeks or fewer of palliative radiotherapy for non-CNS disease, with a 1-week washout, is permitted.

    20. Has received a live vaccine or live-attenuated vaccine within 28 days before the first dose of epcoritamab. Administration of killed vaccines are allowed.

    21. Patients requiring ongoing treatment with warfarin within 7 days of start of epcoritamab

    22. Patients who have received > 140 mg of prednisone for 14 days prior to first dose of epcoritamab or those requiring > 10 mg prednisone (or equivalent) daily, unless needed for disease control.

    23. Confirmed history or current autoimmune disease or other diseases resulting in permanent immunosuppression or requiring permanent immunosuppressive therapy. Low-dose (<=10 mg/day) prednisolone (or equivalent) for rheumatoid arthritis or similar conditions is allowed.

    24. Enrolled on another therapeutic clinical trial for WM at the start of epcoritamab. Concurrent enrollment on another therapeutic clinical trial or any trial designed to impact the efficacy of anti-cancer therapy is prohibited.

    25. Has not adequately recovered after 4 weeks from major surgery or has ongoing surgical complications.

      Note: Biopsy and placement of central venous access devices are not considered major surgery.

    26. Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: N / D
  • Modelo Intervencional: Atribuição de grupo único
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Epcoritamab

Epcoritamab will be administered as consolidative therapy in adults who have been receiving zanubrutinib or ibrutinib.

Step-up epcoritamab dosing during cycle 1, followed by 48 mg every 4 weeks for cycles 2-12

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Rate of complete remission (CR) or very good partial remission (VGPR)
Prazo: 12 cycles (approximately 12 months after treatment begins)

CR: No detectable monoclonal IgM, normal bone marrow morphology with no lymphoplasmacytic lymphoma involvement, and resolution of any baseline extramedullary disease.VGPR: At least a 90% reduction in serum IgM from baseline or a serum IgM level within the normal range.

12 cycles (approximately 12 months after treatment begins)

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Overall survival (OS)
Prazo: From the first dose of epcoritamab until death or up to 5 years after the first dose.

Time from treatment initiation to death from any cause.

From the first dose of epcoritamab until death or up to 5 years after the first dose.
Duration of response (DOR) after BTKi discontinuation
Prazo: After BTKi discontinuation until loss of response, progression, death, or initiation of subsequent therapy.

The length of time that a confirmed CR or VGPR is maintained after BTKi therapy is discontinued.

After BTKi discontinuation until loss of response, progression, death, or initiation of subsequent therapy.
Progression-free survival (PFS)
Prazo: From the first dose of epcoritamab until progression or death.

Time from treatment initiation to disease progression or death from any cause.

From the first dose of epcoritamab until progression or death.
Treatment-emergent adverse events (TEAEs)
Prazo: From the first dose of epcoritamab through 60 days after the last dose, or until new systemic anticancer therapy.

Any new or worsened unfavorable medical occurrence after study treatment begins, regardless of whether it is considered treatment-related.

From the first dose of epcoritamab through 60 days after the last dose, or until new systemic anticancer therapy.
IgM flare
Prazo: Baseline, during treatment, after treatment, and following BTKi discontinuation.

Measured through serial serum IgM.

Baseline, during treatment, after treatment, and following BTKi discontinuation.
Immune effector cell associated neurotoxicity syndrome (ICANS)
Prazo: From the first epcoritamab dose through treatment and safety follow-up, approximately 60 days after the last dose.

ICANs will be assessed using the 10-point ICE tool.

From the first epcoritamab dose through treatment and safety follow-up, approximately 60 days after the last dose.
Cytokine release syndrome (CRS)
Prazo: From the first epcoritamab dose through treatment and safety follow-up, approximately 60 days after the last dose.

CRS will be graded using the ASTCT CRS criteria.

From the first epcoritamab dose through treatment and safety follow-up, approximately 60 days after the last dose.

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Investigadores

  • Investigador principal: Laura S Samples, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Publicações e links úteis

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Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

9 de novembro de 2026

Conclusão Primária (Estimado)

1 de novembro de 2030

Conclusão do estudo (Estimado)

1 de novembro de 2033

Datas de inscrição no estudo

Enviado pela primeira vez

19 de outubro de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

19 de outubro de 2026

Primeira postagem (Real)

20 de outubro de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

4 de novembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

3 de novembro de 2026

Última verificação

15 de outubro de 2026

Mais Informações

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Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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