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EV + Toripalimab vs GC as Neoadjuvant Therapy in Locally Advanced/High-Risk MIBC

9 de junio de 2026 actualizado por: Peking University Third Hospital

A Phase II, Two-arm, Open-label, Multicenter, Randomized Controlled Clinical Study Evaluating the Safety and Efficacy of Enfortumab Vedotin Combined With Toripalimab Versus Gemcitabine Combined With Cisplatin in the Neoadjuvant Treatment of Patients With Locally Advanced/High-risk Muscle-invasive Bladder Cancer

The purpose of this Phase II, open-label, multicenter, randomized controlled study is to evaluate the efficacy and safety of Enfortumab Vedotin in combination with Toripalimab compared to Gemcitabine plus Cisplatin. This regimen is evaluated as a neoadjuvant treatment for patients with locally advanced or high-risk muscle-invasive bladder cancer. Participants will be randomly assigned in a 1:1 ratio to one of two cohorts. Cohort A will receive Enfortumab Vedotin and Toripalimab for 3 treatment cycles. Cohort B will receive Gemcitabine and Cisplatin for 3 treatment cycles. Following the neoadjuvant treatment phase, patients will undergo radical cystectomy and pelvic lymph node dissection. The primary endpoint of the study is the 1-year Event-Free Survival (EFS) rate. Secondary endpoints include pathological downstaging rate (pDR), pathological complete response (pCR), Disease-Free Survival (DFS), Overall Survival (OS), and the assessment of adverse events. Additionally, the study will evaluate the relationship between treatment efficacy and the expression of PD-L1 and Nectin-4 in tumor tissues.

Descripción general del estudio

Tipo de estudio

Intervencionista

Inscripción (Estimado)

58

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

    • Beijing Municipality
      • Beijing, Beijing Municipality, Porcelana, 100191
        • Reclutamiento
        • Peking University Third Hospital
        • Contacto:
          • Clinical Trial Office
          • Número de teléfono: +86 10 8226 5573
          • Correo electrónico: bysy@bjmu.edu.cn

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:

  • Voluntarily agree to participate in the study and sign the informed consent form (ICF).
  • Age ≥ 18 and ≤ 80 years at the time of signing the ICF.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
  • Histologically confirmed muscle-invasive urothelial carcinoma of the bladder, with variant histology components comprising < 50%.
  • Radiographically confirmed non-metastatic urothelial carcinoma (M0). Clinical stage must be locally advanced or possess high-risk features, including at least one of the following: clinical stage cT3-T4aNxM0, or definitive high-risk cT2 (e.g., accompanied by tumor-related hydronephrosis, lymphovascular invasion).
  • Participants must be evaluated as fit for and scheduled to undergo radical surgery, and clinically fit to tolerate cisplatin-based chemotherapy.
  • Able to provide tumor tissue samples (at least 5 unstained slides, or paraffin scrolls/blocks).
  • Expected life expectancy of at least 12 weeks.
  • Adequate organ function, defined by the following laboratory values (obtained without blood transfusion within 14 days or growth factor support within 7 days prior to testing): Absolute neutrophil count (ANC) ≥ 1.5 × 10^9/L; Platelet count (PLT) ≥ 100 × 10^9/L; Hemoglobin (Hb) ≥ 90 g/L; Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 × Upper Limit of Normal (ULN); Total bilirubin (TBIL) ≤ 1.5 × ULN; International Normalized Ratio (INR) ≤ 1.5 × ULN; Creatinine clearance ≥ 60 mL/min (calculated using the Cockcroft-Gault formula); Left ventricular ejection fraction (LVEF) ≥ 50%; QTcF interval ≤ 480 ms.
  • Female participants of childbearing potential must have a negative serum pregnancy test within 7 days prior to the first dose of study treatment and must be willing to use highly effective contraception during the study and for 180 days after the last dose.
  • Male participants with female partners of childbearing potential must be surgically sterile or willing to use highly effective contraception during the study and for 180 days after the last dose.
  • Able to understand and comply with study visits, treatment plans, laboratory tests, and other study procedures.

Exclusion Criteria:

  • Prior systemic anti-tumor therapy for urothelial carcinoma, including radiotherapy, chemotherapy, targeted therapy, or biological therapy (except for intravesical instillation therapy).
  • Prior treatment with PD-1/PD-L1 inhibitors or antibody-drug conjugates (ADCs).
  • Active malignancies other than urothelial carcinoma within 3 years prior to the first dose, except for curatively treated malignancies (e.g., basal or squamous cell skin cancer, localized low-risk prostate cancer, papillary thyroid cancer, carcinoma in situ of the cervix, ductal carcinoma in situ of the breast).
  • Active autoimmune disease requiring systemic treatment (e.g., use of disease-modifying agents, corticosteroids, or immunosuppressants) within 2 years prior to the first dose; or received high-dose steroids (>10 mg/day prednisone equivalent) or other immunosuppressive medications within 14 days prior to the first dose. (Physiological replacement therapies are permitted).
  • Severe thromboembolic events or severe cardiovascular/cerebrovascular diseases within 1 year prior to the first dose, including but not limited to myocardial infarction, unstable angina, pulmonary embolism, cerebral hemorrhage, cerebral infarction, and deep vein thrombosis.
  • Major surgical procedure within 28 days prior to the first dose; or cystoscopy/ureteroscopy biopsy or intravesical therapy within 7 days prior to the first dose.
  • Peripheral neuropathy ≥ Grade 2.
  • Severe dry eye syndrome, active keratitis, corneal ulcers, or conditions assessed by the investigator as increasing the risk of corneal disease and unsuitable for participation.
  • Active infections, including: Positive HBsAg with HBV-DNA copy number ≥ 500 IU/mL; Positive HCV antibody with positive HCV-RNA; Positive HIV antibody; Active tuberculosis infection; Other active infections requiring systemic therapy within 7 days prior to the first dose.
  • Other severe or uncontrolled diseases, including but not limited to: Severe respiratory diseases (e.g., moderate-to-severe interstitial or obstructive pulmonary disease, severe asthma); New York Heart Association (NYHA) Class III or IV heart failure; HbA1c ≥ 8% (except for participants whose fasting blood glucose is stably controlled at ≤ 10 mmol/L with medication); Poorly controlled hypertension (systolic BP ≥ 160 mmHg and/or diastolic BP ≥ 100 mmHg); Large pleural effusion or ascites requiring symptomatic treatment within 14 days prior to the first dose.
  • Receipt of live vaccines within 28 days prior to the first dose or plans to receive live vaccines during the study.
  • Prior allogeneic hematopoietic stem cell transplantation or solid organ transplantation.
  • Use of strong CYP3A4 inhibitors or inducers within 14 days prior to the first dose.
  • Known severe hypersensitivity or intolerance to the study drugs or any of their excipients.
  • Substance abuse or psychiatric disorders that may interfere with study compliance.
  • Any other condition that, in the opinion of the investigator, makes the participant unsuitable for the study.

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: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Experimental: Enfortumab Vedotin + Toripalimab
Participants in this arm will receive neoadjuvant therapy consisting of Enfortumab Vedotin and Toripalimab. Enfortumab Vedotin (1.25 mg/kg) will be administered intravenously on Days 1 and 8 of each 21-day cycle. Toripalimab (240 mg) will be administered intravenously on Day 1 of each 21-day cycle. The treatment will be administered for a total of 3 cycles, followed by radical cystectomy and pelvic lymph node dissection.
Enfortumab Vedotin is administered intravenously at a dose of 1.25 mg/kg on Day 1 and Day 8 of each 21-day cycle for a total of 3 cycles
Toripalimab is administered intravenously at a dose of 240 mg on Day 1 of each 21-day cycle for a total of 3 cycles.
Comparador activo: Active Comparator: Gemcitabine + Cisplatin
Participants in this arm will receive standard neoadjuvant chemotherapy consisting of Gemcitabine and Cisplatin. Gemcitabine (1000 mg/m^2) will be administered intravenously on Days 1 and 8 of each 21-day cycle. Cisplatin (70 mg/m^2) will be administered intravenously on Day 1 of each 21-day cycle. The treatment will be administered for a total of 3 cycles, followed by radical cystectomy and pelvic lymph node dissection.
Gemcitabine is administered intravenously at a dose of 1000 mg/m² on Day 1 and Day 8 of each 21-day cycle for a total of 3 cycles.
Cisplatin is administered intravenously at a dose of 70 mg/m² on Day 1 of each 21-day cycle for a total of 3 cycles.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
1-Year Event-Free Survival (EFS) Rate
Periodo de tiempo: Up to 1 year post-surgery
The percentage of participants who remain free of an event at 1 year. An event is defined as the first occurrence of any of the following: 1) radiographically confirmed disease progression during neoadjuvant therapy (per RECIST v1.1); 2) unresectable tumor or extensive pelvic metastasis identified during surgical exploration; 3) any type of local recurrence or distant metastasis (confirmed radiologically or pathologically) after radical surgery; 4) death from any cause.
Up to 1 year post-surgery

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Pathological Complete Response (pCR) Rate
Periodo de tiempo: At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
The percentage of participants achieving a pathological complete response, defined as the absence of residual tumor in the bladder and lymph nodes (ypT0N0) upon postoperative pathological evaluation.
At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
Pathological Downstaging Rate (pDR)
Periodo de tiempo: At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
The percentage of participants achieving pathological downstaging, defined as pathological stage ≤ ypT1N0 upon postoperative pathological evaluation.
At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
Disease-Free Survival (DFS)
Periodo de tiempo: From the date of radical surgery up to study completion (Assessed every 12 weeks for the first 48 weeks, then every 24 weeks, estimated up to 3 years)
The time from the date of radical surgery to the first documented local recurrence or distant metastasis (identified radiologically or pathologically), or death from any cause, whichever occurs first.
From the date of radical surgery up to study completion (Assessed every 12 weeks for the first 48 weeks, then every 24 weeks, estimated up to 3 years)
Overall Survival (OS)
Periodo de tiempo: From the date of randomization up to study completion (Assessed every 12 weeks, estimated up to 3 years)
The time from the date of randomization to death from any cause.
From the date of randomization up to study completion (Assessed every 12 weeks, estimated up to 3 years)
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From the first dose of study drug up to 28 days after the last dose (or before starting new anti-tumor therapy, whichever is earlier)
Safety will be evaluated by the incidence, severity, and causality of AEs and SAEs. The severity of adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
From the first dose of study drug up to 28 days after the last dose (or before starting new anti-tumor therapy, whichever is earlier)
Biomarker Expression: Nectin-4 and PD-L1
Periodo de tiempo: Baseline (Screening) and at the time of radical surgery
To evaluate the expression levels of Nectin-4 and PD-L1 in tumor tissue samples (obtained at baseline and post-surgery) and to analyze the relationship between these biomarker expressions and clinical efficacy.
Baseline (Screening) and at the time of radical surgery

Otras medidas de resultado

Medida de resultado
Medida Descripción
Periodo de tiempo
Patient-Reported Outcomes (PROs)
Periodo de tiempo: From baseline up to study completion (Estimated up to 36 months)
Patient-reported general cancer quality of life is assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). The EORTC QLQ-C30 is a validated 30-item questionnaire incorporating a Global Health Status/Quality of Life scale, 5 functional scales (physical, role, emotional, cognitive, and social), and multiple symptom scales. All scale scores are linearly transformed to range from 0 to 100. For the Global Health Status and functional scales, higher scores indicate better quality of life or higher functioning. For symptom scales and items, higher scores indicate greater symptom burden.
From baseline up to study completion (Estimated up to 36 months)

Colaboradores e Investigadores

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

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 (Actual)

15 de abril de 2025

Finalización primaria (Estimado)

1 de abril de 2027

Finalización del estudio (Estimado)

1 de diciembre de 2028

Fechas de registro del estudio

Enviado por primera vez

24 de mayo de 2026

Primero enviado que cumplió con los criterios de control de calidad

9 de junio de 2026

Publicado por primera vez (Actual)

15 de junio de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

15 de junio de 2026

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

9 de junio de 2026

Última verificación

1 de mayo de 2026

Más información

Términos relacionados con este estudio

Otros números de identificación del estudio

  • LM2025412
  • IRB00006761 (Otro identificador: Peking University Third Hospital Medical Science Research Ethics Committee)
  • M20250227 (Otro identificador: Peking University Third Hospital Medical Science Research Ethics Committee)

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

INDECISO

Descripción del plan IPD

Individual participant data (IPD) sharing has not been determined at this time. Decisions regarding IPD sharing will be made in accordance with applicable regulations, institutional policies, and sponsor agreements

Información sobre medicamentos y dispositivos, documentos del estudio

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

No

Estudia un producto de dispositivo regulado por la FDA de 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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