EV + Toripalimab vs GC as Neoadjuvant Therapy in Locally Advanced/High-Risk MIBC
2026年6月9日 更新者: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.
調査の概要
状態
募集
研究の種類
介入
入学 (推定)
58
段階
- フェーズ2
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
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Beijing Municipality
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Beijing、Beijing Municipality、中国、100191
- 募集
- Peking University Third Hospital
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コンタクト:
- Clinical Trial Office
- 電話番号:+86 10 8226 5573
- メール:bysy@bjmu.edu.cn
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-
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
説明
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.
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:なし(オープンラベル)
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的: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.
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アクティブコンパレータ: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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この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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1-Year Event-Free Survival (EFS) Rate
時間枠: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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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Pathological Complete Response (pCR) Rate
時間枠: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)
時間枠: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)
時間枠: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)
時間枠: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)
時間枠: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
時間枠: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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その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Patient-Reported Outcomes (PROs)
時間枠: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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協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (実際)
2025年4月15日
一次修了 (推定)
2027年4月1日
研究の完了 (推定)
2028年12月1日
試験登録日
最初に提出
2026年5月24日
QC基準を満たした最初の提出物
2026年6月9日
最初の投稿 (実際)
2026年6月15日
学習記録の更新
投稿された最後の更新 (実際)
2026年6月15日
QC基準を満たした最後の更新が送信されました
2026年6月9日
最終確認日
2026年5月1日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- LM2025412
- IRB00006761 (その他の識別子:Peking University Third Hospital Medical Science Research Ethics Committee)
- M20250227 (その他の識別子:Peking University Third Hospital Medical Science Research Ethics Committee)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
未定
IPD プランの説明
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
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
いいえ
米国FDA規制機器製品の研究
いいえ
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