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 8/26/26
• 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

Recurrent Non-Muscle Invasive Bladder Urothelial Carcinoma; 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

• Pre vs. post intervention urinary biomarkers

  Up to 28 days

Other Outcomes (11)

• 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 new or occasionally recurrent NMIBC (i.e., \</= 2 prior NMIBC episodes in the 18 months preceding the cystoscopy where the index tumor was identified) 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
• Participants with single and multiple tumor lesions
• In the opinion of the treating urologist, the participant is suitable to undergo surgery.
• Participants may have received intravesical chemotherapy at any time.
• 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
• Serum Testosterone \>= 250 ng/dL
• Thyroid stimulating hormone (TSH) within institutional normal
• The effects of bicalutamide on the developing human fetus at the recommended therapeutic dose are unknown. For this reason, men who are having sex must wear a condom when engaging in any activity that allows for passage of ejaculate to another person throughout the course of the study and 4 months after receiving last dose of study intervention. Male participants should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak. Additionally, men must agree to not donate sperm for the purpose of reproduction during the study and for a minimum of 4 months after receiving the last dose of study intervention
• Ability to understand and the willingness to sign a written informed consent document

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.
• 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 4 months after the last dose of study intervention.
• Participants with suspected Carcinoma in Situ (CIS) without suspected Ta or T1 disease are not eligible.
• Participants with a history of documented CIS within 2 years are not eligible.
• Participants who have received Gacillus Calmette-Guerin (BCG) within 2 years are not eligible.

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

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

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: June 25, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: September 30, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share

Locations

US (6)