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

10 de septiembre de 2026 actualizado 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.

Descripción general del estudio

Estado

Aún no reclutando

Tipo de estudio

Intervencionista

Inscripción (Estimado)

39

Fase

  • Fase 2

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Clinical complete response rate
Periodo de tiempo: 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
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Event-free survival
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Investigadores

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

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de diciembre de 2026

Finalización primaria (Estimado)

1 de febrero de 2029

Finalización del estudio (Estimado)

1 de octubre de 2030

Fechas de registro del estudio

Enviado por primera vez

3 de junio de 2026

Primero enviado que cumplió con los criterios de control de calidad

8 de junio de 2026

Publicado por primera vez (Actual)

11 de junio de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

14 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

10 de septiembre de 2026

Última verificación

1 de septiembre de 2026

Más información

Términos relacionados con este estudio

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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