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A Phase 1 Dose-escalation Study of UGN-501 Administered Intravesically in Adult Participants With Recurrent NMIBC

2026年7月21日 更新者：UroGen Pharma, Inc., a subsidiary of UroGen Pharma Ltd.

A Phase 1, Open-label, Dose-escalation Study to Investigate the Safety, Tolerability, and Pharmacokinetics of UGN-501 Administered Intravesically in Adult Participants With Recurrent Non-muscle Invasive Bladder Cancer (NMIBC)

This study is being conducted to evaluate the safety and tolerability of UGN-501 administered intravesically in adult participants with recurrent non-muscle invasive bladder cancer (NMIBC) and to determine the recommended Phase 2 dose (RP2D).

研究概览

地位

尚未招聘

条件

干预/治疗

详细说明

This is a Phase 1, open-label, dose-escalation, multicenter study to investigate the safety, tolerability, immunogenicity, and pharmacokinetics (PK) of UGN-501, a chimeric oncolytic adenovirus, administered intravesically in participants with recurrent NMIBC.

Eligible participants will enter a 12-week Induction Period. Participants with Ta and/or T1 disease who do not have disease recurrence, and participants with carcinoma in situ (CIS) who have a complete response (CR) at Week 12, will enter the Maintenance Period. Disease assessments will be performed every 3 months through Month 15, or until disease recurrence, disease progression, or death, whichever occurs first.

The maximum duration of participation is approximately 15 months.

This master protocol may include multiple study intervention arms designed to independently evaluate UGN-501. Any additional study intervention arms or dose expansions will be added by protocol amendment.

研究类型

介入性

注册 (估计的)

30

阶段

  • 阶段1

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 美国
    • Arizona
      • Queen Creek、Arizona、美国、85140
        • East Valley Urology Center of Arizona
        • 首席研究员：
          • Harpreet Wadhwa, MD
        • 接触：
    • California
      • Bakersfield、California、美国、93301
      • Murrieta、California、美国、92563
        • Urology Center of Southern California
        • 首席研究员：
          • Madhumitha Reddy, DO
        • 接触：
    • Massachusetts
      • Boston、Massachusetts、美国、02115
        • Brigham and Women's Hospital
        • 接触：
        • 首席研究员：
          • Matthew Mossanen, MD, PhD
    • Nebraska
      • Omaha、Nebraska、美国、68198
        • University of Nebraska Medical Center
        • 首席研究员：
          • Jared Schober, MD
        • 接触：
    • New York
      • New York、New York、美国、10029
        • Ichan School of Medicine at Mount Sinai
        • 首席研究员：
          • John Sfakianos, MD
        • 接触：
      • Syracuse、New York、美国、13210
        • SUNY Upstate Medical University
        • 首席研究员：
          • Joseph Jacob, MD
        • 接触：
    • Pennsylvania
      • Philadelphia、Pennsylvania、美国、19104
        • University of Pennsylvania-Perelman Center for Advanced Medicine
        • 首席研究员：
          • Trinity Bivalacqua, MD, PhD
        • 接触：
    • South Carolina
      • Myrtle Beach、South Carolina、美国、29572
        • START Carolinas
        • 首席研究员：
          • Abhishek Srivastava, MD
        • 接触：
    • Texas
      • Arlington、Texas、美国、76017
        • UPNT Research Institute
        • 接触：
        • 首席研究员：
          • Patrick Collini, MD
      • Austin、Texas、美国、78759

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  1. Participant must be 18 years of age or older at the time of signing informed consent.
  2. Has confirmed recurrent non-muscle invasive bladder cancer (NMIBC) with high-grade Ta and/or T1 disease and/or carcinoma in situ (CIS), or recurrent low-grade Ta and/or T1 disease.

  3. Participants with high-grade Ta and/or T1 disease and/or CIS must meet one of the following criteria:

    • Has BCG-unresponsive disease, defined as:
    • persistent or recurrent CIS alone or with recurrent Ta/T1 disease within 12 months of completion of adequate BCG therapy; or
    • recurrent high-grade Ta/T1 disease within 6 months of completion of adequate BCG therapy; or
    • high-grade T1 disease at the first evaluation following a BCG induction course.

    Adequate BCG therapy is defined as at least 5 of 6 doses of an initial induction course plus either at least 2 of 3 doses of maintenance therapy or at least 2 of 6 doses of a second induction course. Participants with BCG-unresponsive disease also must be unwilling or unfit to undergo radical cystectomy.

    • Is BCG-exposed, defined as high-grade persistent or recurrent NMIBC within 24 months of the last dose of BCG but not meeting the definition of BCG-unresponsive disease, and received at least 5 of 6 doses of an initial induction course of BCG.
    • Is BCG intolerant, defined as the inability to tolerate at least 1 full induction course of BCG.
    • Has a high-grade Ta tumor ≤3 cm and failed at least 1 previous course of therapy, such as TURBT plus an adjuvant induction course of intravesical chemotherapy.

  4. Participants with low-grade disease must meet one of the following criteria:

    • Ta tumors recurring within 1 year.
    • Ta solitary tumor >3 cm.
    • Ta multifocal tumors.
    • T1 tumors.
  5. All visible papillary tumors must be resected and obvious areas of CIS fulgurated during Screening or within 6 weeks before Screening. Participants with T1 disease should have a re-staging TURBT during Screening or within 6 weeks before Screening. Enhanced cystoscopy, such as blue light cystoscopy or other locally accepted modalities, is permitted, but the same modality must be used for all subsequent disease assessments.
  6. Has Eastern Cooperative Oncology Group performance status score ≤2.
  7. Has no concomitant upper tract urothelial carcinoma (UTUC) or urothelial carcinoma within the prostatic stroma. Freedom from upper tract disease, if clinically indicated, must be demonstrated by no evidence of upper tract tumor by either IV pyelogram, retrograde pyelogram, CT urogram with or without contrast, MRI urogram with or without contrast, or ureteroscopy with ureteral washing for cytology, performed within 6 months of enrollment. Participants with urothelial carcinoma involving the prostatic urethra, where carcinoma is confined to the ducts and/or epithelium, may be included after a restaging TURBT is performed to rule out more extensive disease involving the prostatic stroma.
  8. Participants with prostate cancer may be eligible if, after surgery or radiation, they do not meet criteria for biochemical recurrence, or if they are on active surveillance at very low, low, or favorable risk for progression, defined as Gleason Grade Group 1 or 2, Gleason score ≤7, prostate-specific antigen <20 ng/dL, and cT1-cT2b, at the discretion of the investigator.

  9. Has adequate organ and bone marrow function within 14 days of treatment initiation, as determined by routine laboratory tests:

    • Leukocytes ≥3000/μL.
    • Absolute neutrophil count ≥1500/μL.
    • Platelets ≥100,000/μL.
    • Hemoglobin ≥9.0 g/dL.
    • Total bilirubin ≤1.5 × upper limit of normal (ULN).
    • Aspartate aminotransferase (AST) ≤2.5 × UL
    • Alanine aminotrasferase (ALT) ≤2.5 × ULN.
    • Alkaline phosphatase ≤2.5 × ULN.
    • Estimated creatinine clearance ≥30 mL/min using the Cockcroft-Gault equation.
  10. Has a life expectancy >12 months.
  11. Participants and participant partners must agree to follow contraception and barrier method requirements consistent with local regulations and the protocol during the study intervention period and for at least 12 weeks after the last study intervention instillation.
  12. Has signed informed consent and is willing and able to comply with the requirements and restrictions listed in the informed consent form and protocol.

Exclusion Criteria:

  1. Current or previous evidence of muscle invasive, locally advanced nonresectable, or metastatic urothelial carcinoma, including T2, T3, T4, and/or stage IV disease.
  2. Current systemic therapy for bladder cancer.
  3. Prior treatment with any human adenovirus-based therapy, such as nadofaragene firadenovec-vncg or cretostimogene grenadenorepvec.
  4. Intravesical therapy within 4 weeks before starting study intervention, including but not limited to BCG, chemotherapy, and nogapendekin alfa inbakicept.
  5. Participation in a study of an investigational agent with receipt of study therapy, or receipt of an investigational device, within 4 weeks before the first dose of study intervention.
  6. Receipt of immune modulator therapy within 5 half-lives of starting study intervention, including but not limited to pembrolizumab, BCG, and nogapendekin alfa inbakicept.
  7. Receipt of a vaccine or anti-viral agent within 2 weeks before starting study intervention.
  8. Active infection requiring systemic therapy, including urinary tract infection. Participants may enter the study once the infection is satisfactorily treated.
  9. Immunocompromised state, including but not limited to HIV infection or any condition that required systemic immunosuppressive treatment in the past 2 years, such as active systemic autoimmune disease or solid organ or stem cell transplant. Short courses (≤14 days) of steroids for medical reasons without anticancer intent, such as atopic dermatitis, psoriasis, infection, or allergic reaction, are permitted if the last dose was at least 4 weeks before the first dose of study intervention.
  10. Any medical, psychological, familial, sociological, or geographical condition that, in the opinion of the investigator, would preclude participation in the study.
  11. History of malignancy of another organ system within the past 5 years, except previously treated UTUC, basal cell carcinoma or squamous cell carcinoma of the skin, and/or prostate cancer meeting protocol-specified criteria.
  12. Cannot tolerate intravesical dosing or intravesical surgical manipulation.
  13. Known allergy or hypersensitivity to any of the study interventions or any study intervention excipients.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：非随机化
  • 介入模型：顺序分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：UGN-501 Dose Escalation (Part 1)
Dose escalation of UGN-501 in participants with recurrent NMIBC with high grade (HG) Ta and/or T1 disease and/or CIS or recurrent low grade (LG) Ta and/or T1 disease.
生物：UGN-501
UGN-501 is administered by intravesical instillation into the bladder. Participants receive 6 once-weekly instillations during the Induction Period. Participants eligible for maintenance receive 3 once-weekly instillations quarterly from Month 3 through Month 12.

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs)
大体时间：Up to 15 Months
The number of participants with each type of event will be summarized.
Up to 15 Months
Concentration of UGN-501 in blood and urine
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks
Complete response rate (CRR)
大体时间：3 Months
CRR is defined as the proportion of CIS participants who achieved CR at the Week 12 (3-month) Visit.
3 Months
Recurrence-free survival (RFS) rate
大体时间：6 Months
RFS rate is defined as the proportion of participants with Ta/T1 disease who are recurrence-free at the 6-month Visit.
6 Months

次要结果测量

结果测量
措施说明
大体时间
Presence of anti-drug antibodies (ADA) in serum
大体时间：Up to 12 Weeks
The number of participants with ADA will be summarized.
Up to 12 Weeks
UGN-501 maximum concentration (Cmax) following single and repeat dose administration
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics
Up to 12 Weeks
UGN-501 area under the concentration-time curve (AUC) following single and repeat dose administration
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks
UGN-501 terminal half-life (t1/2) following single and repeat dose administration
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks
UGN-501 time to maximum concentration (tmax) following single and repeat dose administration
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks
UGN-501 concentration at the end of a dosing interval (Ctau) following single and repeat dose administration
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks
Evaluation of viral shedding in urine following singe and repeat dose administration.
大体时间：Up to 12 Weeks
Data will be summarized using descriptive statistics.
Up to 12 Weeks

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

UroGen Pharma, Inc., a subsidiary of UroGen Pharma Ltd.

调查人员

  • 研究主任：Sebastian Mirkin, MD、UroGen Pharma

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年9月30日

初级完成 (估计的)

2029年8月2日

研究完成 (估计的)

2029年8月2日

研究注册日期

首次提交

2026年7月21日

首先提交符合 QC 标准的

2026年7月21日

首次发布 (实际的)

2026年7月24日

研究记录更新

最后更新发布 (实际的)

2026年7月24日

上次提交的符合 QC 标准的更新

2026年7月21日

最后验证

2026年7月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • UGN-501-101

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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