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Neoadjuvant Enfortumab Vedotin Plus Pembrolizumab in Muscle-Invasive Bladder Cancer and Upper Tract Urothelial Carcinoma

10 de setembro de 2026 atualizado por: University of Florida

Radical cystectomy remains the standard curative-intent treatment for most patients with muscle-invasive bladder cancer, but it is associated with significant morbidity and long-term quality-of-life implications. Trimodality therapy is an accepted standard-of-care alternative for carefully selected patients who wish to preserve their bladder; however, optimal patient selection remains challenging.

The combination of enfortumab vedotin plus pembrolizumab (EV-P) has demonstrated remarkable activity in urothelial carcinoma, including in the perioperative setting, with pathologic complete response rates of approximately 50-60%. These results generate the hypothesis that a subset of patients may achieve sufficiently deep responses to allow selective deferral of cystectomy. Cohort A of this trial prospectively evaluates the use of multimodal response assessment (pelvic MRI and TURBT, ctDNA) to guide individualized decisions regarding cystectomy versus bladder preservation.

Radical nephroureterectomy (RNU) remains the standard curative-intent treatment for high-risk upper tract urothelial carcinoma (UTUC), but recurrence rates after surgery alone are high. Neoadjuvant cisplatin-based chemotherapy improves pathologic outcomes and is supported by phase II data, but its delivery is constrained by baseline renal dysfunction and the further decline in glomerular filtration that follows RNU - historically, only about 20% of patients remain cisplatin-eligible postoperatively, which is the principal rationale for delivering platinum in the neoadjuvant rather than adjuvant setting. A large fraction of patients with UTUC are cisplatin-ineligible at baseline, and no level 1 evidence supports a specific neoadjuvant regimen in this population.

EV-P is not constrained by renal function and has produced unprecedented activity in urothelial carcinoma. In the EV-302 upper tract subgroup, EV-P achieved an objective response rate of 67.7% and a complete response rate of 28.6%, with survival benefit preserved relative to platinum-based chemotherapy. In the perioperative bladder cancer setting, EV-P has yielded pathologic complete response rates of approximately 50-60%. However, available data on EV-P in UTUC are restricted to the metastatic setting, and prospective evaluation in the neoadjuvant setting is lacking. Cohort B of this trial addresses this gap by prospectively evaluating neoadjuvant EV-P followed by RNU in patients with high-risk UTUC, with pathologic complete response as the primary endpoint.

Visão geral do estudo

Status

Ainda não está recrutando

Tipo de estudo

Intervencional

Inscrição (Estimado)

39

Estágio

  • Fase 2

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

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Descrição

Inclusion Criteria:

  • Adults ≥ 18 years of age.
  • ECOG Performance Status of 0-2
  • For Cohort A, a clinical or pathological diagnosis consistent with muscle-invasive urothelial carcinoma of the bladder (locoregional), with a clinical staging of T2 or higher, N0 to N2, and M0.

For Cohort B, a clinical or pathological diagnosis of upper tract urothelial carcinoma, with high-intermediate risk or higher per NCCN guidelines, planned for definitive surgery.

  • High-intermediate: High grade + Unifocal <1.5 cm
  • High: High grade + Multifocal or >1.5 cm or high-grade renal obstruction or invasion on axial imaging Any proportion of divergent differentiation is permitted across all histologies (except neuroendocrine - see exclusion criteria), as long as a urothelial component is identified.

    • Eligible to receive enfortumab vedotin plus pembrolizumab (EV-P) in the opinion of the investigators.
    • Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures.
    • Subjects must not have more than one active malignancy at the time of enrollment.

Note: Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen or primary endpoint (as determined by the treating physician) are eligible for this trial.

- Subjects of childbearing potential (SOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for at least 4 months after the last dose of EV-P to minimize the risk of pregnancy. Prior to study enrollment, subjects of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy.

SOCBP includes any subject who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or who is not post-menopausal. Post-menopause is defined as:

  • Amenorrhea that has lasted for ≥ 12 consecutive months without another cause, or
  • For subjects with irregular menstrual periods who are taking hormone replacement therapy (HRT), a documented serum follicle-stimulating hormone (FSH) level of greater than 35 mIU/mL.

    • Subjects with partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 4 months following the last dose of EV-P.

Exclusion Criteria:

  • Histology that contains any component of neuroendocrine carcinoma.
  • Any known contraindications to enfortumab vedotin plus pembrolizumab.
  • Subjects who are confirmed to be pregnant or breastfeeding.
  • History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.
  • Administration of a vaccine containing live virus within 30 days prior to the first dose of trial treatment. Note: Most flu vaccines are killed viruses, with the exception of the intra-nasal vainer (Flu-Mist) which is an attenuated live virus and therefore prohibited for 30 days prior to first dose.
  • Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.

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: Não randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Cohort A (neoadjuvant EV-P + response-adapted management)

Following neoadjuvant EV-P, participants in this cohort will have response-adapted management based on if they achieved a composite clinical complete response (cCR) to determine the next step in their treatment on this study. A participant has achieved cCR if ALL the following are met:

  1. ctDNA is negative,
  2. Imaging are negative for lymph nodes and distant metastases, and
  3. TURBT shows T0 or Ta low-grade only, and a negative urinary cytology.

If a participant achieves cCR, they will have a choice between:

  • receive 5 more cycles of EV-P, followed by pembrolizumab alone to complete 1 year of therapy.
  • Alternatively, if participant wishes, partial or radical cystectomy.

If a participant does not achieve cCR, they will have cystectomy.

Participants in both cohorts will first receive neoadjuvant enfortumab vedotin + pembrolizumab (EV-P) for 4 cycles. Each cycle is 21 days. Participants will be given 1.25 mg/kg enfortumab vedotin intravenously on days 1 and 8 and 200 mg pembrolizumab intravenously on day 1 of each cycle. Participants in Cohort A may then choose to receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy if they have a composite complete clinical response. Participants in Cohort B will then receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy following their definitive surgery.
Experimental: Cohort B (neoadjuvant EV-P + surgery + adjuvant EV-P)
Participants in both cohorts will first receive neoadjuvant enfortumab vedotin + pembrolizumab (EV-P) for 4 cycles. Each cycle is 21 days. Participants will be given 1.25 mg/kg enfortumab vedotin intravenously on days 1 and 8 and 200 mg pembrolizumab intravenously on day 1 of each cycle. Participants in Cohort A may then choose to receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy if they have a composite complete clinical response. Participants in Cohort B will then receive 5 more cycles of EV-P followed by pembrolizumab alone to complete 1 year of study therapy following their definitive surgery.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Clinical complete response rate
Prazo: 3 weeks following completion of neoadjuvant therapy

Determine the percent of subjects in Cohort A that achieve a clinical complete response following completion of neoadjuvant EV-P. A clinical complete response is defined as having all three of the following:

  • A negative ctDNA result,
  • Imaging of the pelvis with negative lymph nodes and no distant metastasis on CT scans, AND
  • A TURBT showing T0 or low-grade Ta only, and a negative urinary cytology.
3 weeks following completion of neoadjuvant therapy
Pathologic complete response rate
Prazo: 8 weeks following completion of neoadjuvant therapy
Determine the percent of subjects in Cohort B who achieve a pathologic complete response at the time of surgery. A pathologic complete response is defined as having disease staging of pT0N0 at time of surgery.
8 weeks following completion of neoadjuvant therapy

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Event-free survival
Prazo: 1 year from the start of EV-P
Determine the event-free survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Event-free survival
Prazo: 2 years from the start of EV-P
Determine the event-free survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Overall survival
Prazo: 1 year from the start of EV-P
Determine the overall survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Overall survival
Prazo: 2 years from the start of EV-P
Determine the overall survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Bladder-intact survival
Prazo: 1 year from the start of EV-P
Determine the bladder-intact survival of patients in Cohort A at 1 year from the start of EV-P
1 year from the start of EV-P
Bladder-intact survival
Prazo: 2 years from the start of EV-P
Determine the bladder intact survival of patients in Cohort A at 2 years from the start of EV-P
2 years from the start of EV-P
Pathological complete response
Prazo: After completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of subjects in cohort A who achieve a pathological complete response after first 4 cycles of EV-P.
After completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Pathologic downstaging
Prazo: At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of patients in cohort A who achieve pathological downstaging (disease staging <pT2N0) after the first 4 cycles of EV-P
At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Event-free survival
Prazo: 2 years from the start of EV-P
Determine the event-free survival of patients in Cohort B at 2 years from the start of EV-P
2 years from the start of EV-P
Overall survival
Prazo: 2 years from the start of EV-P
Determine the overall survival of patients in Cohort B at 2 years from the start of EV-P
2 years from the start of EV-P
Pathologic downstaging
Prazo: At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)
Determine number of patients in cohort B who achieve pathological downstaging (disease staging <pT2N0) after the first 4 cycles of EV-P
At completion of first 4 cycles of EV-P (each cycle of EV-P is 21 days)

Colaboradores e Investigadores

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

Patrocinador

Investigadores

  • Investigador principal: Daniel Araujo, MD, University of Florida

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)

1 de dezembro de 2026

Conclusão Primária (Estimado)

1 de fevereiro de 2029

Conclusão do estudo (Estimado)

1 de outubro de 2030

Datas de inscrição no estudo

Enviado pela primeira vez

3 de junho de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

8 de junho de 2026

Primeira postagem (Real)

11 de junho de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

14 de setembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

10 de setembro de 2026

Última verificação

1 de setembro de 2026

Mais Informações

Termos relacionados a este estudo

Informações sobre medicamentos e dispositivos, documentos de estudo

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Sim

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Não

produto fabricado e exportado dos EUA

Não

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