A Randomized Trial of Bicalutamide in Non-Muscle Invasive Bladder Cancer

NCT05521698 | Recruiting Phase 1 DMC FDA Drug

• 6 other identifiers: 2026-0131P30CA014520UG1CA242635NCI-2022-06871INT22-09-01Protocol Version 12/23/25
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 6

Brief Summary

This phase I trial evaluates the effects of bicalutamide, compared to no study drug (NSD), on epidermal growth factor receptor (EGFR) protein expression in patients with non-muscle invasive bladder cancer. Bicalutamide is in a class of medications called androgen receptor inhibitors. It works by blocking the effects of androgen (a male reproductive hormone) to stop the growth and spread of tumor cells. Previous studies have suggested that expression of a protein called EGFR on tumor cells is related to bladder cancer disease progression. This trial may help doctors evaluate if bicalutamide has any effect on EGFR expression in patients with non-muscle invasive bladder cancer.

Conditions

Non-Muscle Invasive Bladder Urothelial Carcinoma; Recurrent Non-Muscle Invasive Bladder Urothelial Carcinoma

Outcome Measures

Primary Outcomes (1)

• Average Epidermal Growth Factor Receptor (EGFR) expression level

  Will be analyzed as a continuous variable. A two-sample t-test will be conducted to test whether there are significant differences of the log-transformed EGFR expression level (measured by reverse transcriptase polymerase chain reaction \[rtPCR\]) in normal appearing urothelium adjacent to tumor in participants treated with anti-androgen therapy versus (vs.) NSD participants. In case the normality assumption of the two-sample t-test does not hold, Wilcoxon rank-sum test will be performed as a sensitivity analysis. Considering androgen receptor (AR) status can be a treatment effect modifier, a regression analysis will also be performed with the log-transformed EGFR expression level as the outcome and treatment status (bicalutamide or NSD), AR status, and treatment-AR interaction as the predictors.

  Up to 28 days

Secondary Outcomes (3)

• Effect of bicalutamide on EGFR expression

  Up to 28 days

• AR expression in adjacent urothelium

  Up to 28 days

• Toxicity of treatment

  Up to 28 days

Other Outcomes (14)

• AR and EGFR (and possibly phosphorylated EGFR [pEGFR]) staining levels

  Up to 28 days

• Expression of direct androgen response gene (ADAR)-2

  Up to 28 days

• Ki-67 expression

  Up to 28 days

Study Arms (2)

Arm 1 (bicalutamide,TURBT)

EXPERIMENTAL

Patients receive bicalutamide PO QD on days 1-21. Patients undergo TURBT on day 21. Up to 28 days of bicalutamide prior to TURBT is permitted in the absence of unacceptable toxicity. Patients undergo blood and urine sample collection throughout the study. Patients may optionally undergo tumor biopsy at baseline.

Drug: Bicalutamide; Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Other: Questionnaire Administration; Procedure: Transurethral Resection of Bladder Tumor

Arm 2 (TURBT)

EXPERIMENTAL

Patients undergo TURBT on day 21. Patients undergo blood and urine sample collection throughout the study. Patients may optionally undergo tumor biopsy at baseline.

Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Other: Questionnaire Administration; Procedure: Transurethral Resection of Bladder Tumor

Interventions

Biopsy Procedure PROCEDURE

Undergo tumor biopsy

Also known as: Biopsy, BIOPSY_TYPE, Bx

Arm 1 (bicalutamide,TURBT); Arm 2 (TURBT)

Questionnaire Administration OTHER

Ancillary studies

Arm 1 (bicalutamide,TURBT); Arm 2 (TURBT)

Transurethral Resection of Bladder Tumor PROCEDURE

Undergo TURBT

Also known as: Transurethral resection (TURBT), TURBT

Arm 1 (bicalutamide,TURBT); Arm 2 (TURBT)

Biospecimen Collection PROCEDURE

Undergo blood and urine sample collection

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Arm 1 (bicalutamide,TURBT); Arm 2 (TURBT)

Bicalutamide DRUG

Given PO

Also known as: Casodex

Arm 1 (bicalutamide,TURBT)

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Biologic male adults (\>= 18 years old)
• Note: Because no dosing or adverse event (AE) data are currently available on the use of bicalutamide in participants \< 18 years of age, children are excluded from this study but will be eligible for future pediatric trials, if applicable
• Have suspected non-muscle invasive bladder carcinoma (NMIBC) on clinic-based cystoscopy or imaging as viewed by an American Urological Association (AUA) board-certified urologist
• Have had cross sectional imaging of the abdomen and pelvis (computed tomography \[CT\] or magnetic resonance imaging \[MRI\] with or without contrast) within 6 months prior to enrollment with no signs of upper tract urothelial cancer (UC), invasive, nor metastatic disease
• Note: If adenopathy or upper tract abnormalities are identified, a negative biopsy and or ureteroscopy is required prior to enrollment
• Newly suspected, diagnosed, or occasionally recurrent bladder cancer (BC)
• Note: Occasional recurrence is defined as =\< 2 prior NMIBC episodes in the 18 months preceding cystoscopy where the index tumor was identified
• Participants with single and multiple tumor lesions
• Eastern Cooperative Oncology Group (ECOG) performance status =\< 1
• Total bilirubin =\< 1.5 x institutional upper limit of normal (note: in participants with Gilbert's syndrome, if total bilirubin is \> 1.5 x upper limit of normal, measure direct and indirect bilirubin and if direct bilirubin is =\< 1.5 x upper limit of normal, participants may be eligible)
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) =\< 2 × institutional upper limit of normal
• Alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) =\< 2 × institutional upper limit of normal
• Urine Culture \< 50,000 colonies/cc of 1 or more organisms (if found and treated and a confirmed negative culture obtained off antibiotics before study drug is started, they will be eligible)
• Serum Testosterone \>= 250 ng/dL
• Thyroid stimulating hormone (TSH) within institutional normal

You may not qualify if:

• Participants who have had a previous exposure to sex hormone (e.g., exogenous androgens) or anti-androgenic therapies (e.g., luteinizing hormone-releasing hormone \[LHRH\] agonists, LHRH antagonists, 5 alpha reductase-inhibitors, abiraterone or other anti-androgens) within 6 months of accrual
• Participants taking Coumarin derivative anticoagulation (e.g., warfarin). Other anticoagulation medications are allowed.
• Participants receiving any other investigational agents.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to bicalutamide.
• History of prior or concurrent muscle invading UC, or concurrent prostatic urethral, urethral, or upper tract UC or non-urothelial bladder cancer
• History of radiation therapy to the pelvis, prostate or prostatic bed, or rectum
• Any condition (uncontrolled intercurrent illness, psychiatric illness, or social situation) for which, in the opinion of the investigator, participation would not be in the best interest of the participant (e.g., compromise the well-being) or that could prevent, limit, or confound the protocol-specified assessments.
• Participants with severe or unstable angina, myocardial infarction, symptomatic congestive heart failure, uncontrolled hypertension, recent arterial or venous thromboembolic events (e.g. pulmonary embolism, cerebrovascular accident including transient ischemic attacks) for which anticoagulation therapy is ongoing, or clinically significant ventricular arrhythmias.
• In the opinion of the investigator, participant has underlying uncontrolled hypertension, high cholesterol, or diabetes.
• Allergy or hypersensitivity to bicalutamide, or excipients, unable or unwilling to take ADT.
• Plans to father a child while enrolled in this study or within 130 days after the last dose of study intervention.

Sponsors & Collaborators

• National Cancer Institute (NCI): collaborator
• University of Wisconsin, Madison: lead

Study Sites (6)

University of Arizona Cancer Center - Prevention Research Clinic

Tucson, Arizona, 85719, United States

NOT YET RECRUITING

Cedars Sinai Medical Center

Los Angeles, California, 90048, United States

NOT YET RECRUITING

National Cancer Institute Urologic Oncology Branch

Bethesda, Maryland, 20892, United States

NOT YET RECRUITING

University of Rochester

Rochester, New York, 14642, United States

NOT YET RECRUITING

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

NOT YET RECRUITING

University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States

RECRUITING

MeSH Terms

Interventions

bicalutamide; Biopsy; Specimen Handling; Transurethral Resection of Bladder

Intervention Hierarchy (Ancestors)

Cytodiagnosis; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Diagnostic Techniques, Surgical; Surgical Procedures, Operative; Investigative Techniques; Urologic Surgical Procedures; Urogenital Surgical Procedures

Study Officials

• Edward M Messing

  University of Wisconsin, Madison

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The participants and the study team will be blinded.
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2022
• First Posted: August 30, 2022
• Study Start: May 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (6)