- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT07647289
EV + Toripalimab vs GC as Neoadjuvant Therapy in Locally Advanced/High-Risk MIBC
9 de junho de 2026 atualizado 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.
Visão geral do estudo
Status
Recrutamento
Tipo de estudo
Intervencional
Inscrição (Estimado)
58
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.
Locais de estudo
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Beijing Municipality
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Beijing, Beijing Municipality, China, 100191
- Recrutamento
- Peking University Third Hospital
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Contato:
- Clinical Trial Office
- Número de telefone: +86 10 8226 5573
- E-mail: bysy@bjmu.edu.cn
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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:
- 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.
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: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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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.
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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.
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Comparador Ativo: 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.
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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.
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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1-Year Event-Free Survival (EFS) Rate
Prazo: Up to 1 year post-surgery
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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.
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Up to 1 year post-surgery
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Pathological Complete Response (pCR) Rate
Prazo: At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
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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.
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At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
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Pathological Downstaging Rate (pDR)
Prazo: At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
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The percentage of participants achieving pathological downstaging, defined as pathological stage ≤ ypT1N0 upon postoperative pathological evaluation.
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At the time of radical surgery (Expected to be within 4-6 weeks after the last cycle of neoadjuvant therapy)
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Disease-Free Survival (DFS)
Prazo: 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)
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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.
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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)
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Overall Survival (OS)
Prazo: From the date of randomization up to study completion (Assessed every 12 weeks, estimated up to 3 years)
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The time from the date of randomization to death from any cause.
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From the date of randomization up to study completion (Assessed every 12 weeks, estimated up to 3 years)
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Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: 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)
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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.
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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)
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Biomarker Expression: Nectin-4 and PD-L1
Prazo: Baseline (Screening) and at the time of radical surgery
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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.
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Baseline (Screening) and at the time of radical surgery
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Outras medidas de resultado
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Patient-Reported Outcomes (PROs)
Prazo: From baseline up to study completion (Estimated up to 36 months)
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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.
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From baseline up to study completion (Estimated up to 36 months)
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
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 (Real)
15 de abril de 2025
Conclusão Primária (Estimado)
1 de abril de 2027
Conclusão do estudo (Estimado)
1 de dezembro de 2028
Datas de inscrição no estudo
Enviado pela primeira vez
24 de maio de 2026
Enviado pela primeira vez que atendeu aos critérios de CQ
9 de junho de 2026
Primeira postagem (Real)
15 de junho de 2026
Atualizações de registro de estudo
Última Atualização Postada (Real)
15 de junho de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
9 de junho de 2026
Última verificação
1 de maio de 2026
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Neoplasias
- Neoplasias por Tipo Histológico
- Neoplasias Glandulares e Epiteliais
- Carcinoma
- Carcinoma de Células de Transição
- Compostos heterocíclicos, 1 anel
- Compostos heterocíclicos
- Terapêutica
- Produtos químicos inorgânicos
- Compostos de cloro
- Compostos de nitrogênio
- Desoxicitidina
- Citidina
- Nucleosídeos de pirimidina
- Pirimidinas
- Compostos de platina
- Gemcitabina
- Cisplatina
- Toripalimab
- Terapia medicamentosa
- Enitumab Vedotin
Outros números de identificação do estudo
- LM2025412
- IRB00006761 (Outro identificador: Peking University Third Hospital Medical Science Research Ethics Committee)
- M20250227 (Outro identificador: Peking University Third Hospital Medical Science Research Ethics Committee)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
INDECISO
Descrição do plano 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
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Não
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .