Testing the Effects of Low Dose Apalutamide on Prostate-Specific Antigen (PSA) Levels in Men Scheduled for Removal of the Prostate Gland

NCT04530552 | Active Not Recruiting Phase 2 Results DMC FDA Drug

• 5 other identifiers: NCI-2020-063222102475889UAZ20-01-01P30CA023074UG1CA242596
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 5

Brief Summary

Apalutamide is an anti-androgen that blocks the effect of testosterone on prostate cancer growth. This phase IIa trial is designed to determine whether very low doses of apalutamide, given for 3 to 4 weeks before prostate surgery to men with prostate cancer confined to the prostate gland, reduces plasma levels of PSA (a biomarker of apalutamide's ability to block testosterone). If low dose apalutamide lowers PSA levels in this setting, further study of this agent in men with localized prostate cancer who wish to delay definitive therapy with surgery or radiation may be warranted.

Conditions

Localized Prostate Carcinoma; Stage I Prostate Cancer AJCC v8; Stage II Prostate Cancer AJCC v8; Prostate Adenocarcinoma

Outcome Measures

Primary Outcomes (1)

• Change in Prostate Specific Antigen (PSA) Levels

  The proportion of participants with \>= 25% decline in PSA levels (from baseline to end-of-intervention) will be reported along with the 97.5% credible interval for the response rate based on the posterior distribution of the response rate derived from a non-informative prior for the response rate, which is consistent with the Bayesian approach.

  Baseline to end-of-intervention (mean 4.8 weeks; up to 9 weeks)

Secondary Outcomes (3)

• Reversibility of Testosterone Levels

  End-of-intervention to post-operation (mean 2.7 days; up to 7 days)

• Post-intervention Plasma Trough Apalutamide Concentrations

  End-of-intervention (mean 4.8 weeks; up to 9 weeks)

• Health-related Quality of Life (HRQOL)

  Baseline to end of intervention (mean 4.8 weeks; up to 9 weeks)

Other Outcomes (3)

• Gleason Score of Pre- and Post-intervention Tumor(s) With Matched Location

  Up to 7-14 days after prostate surgery

• Intra-prostatic Immune Cell Infiltration

  Up to 7-14 days after prostate surgery

• Effects of Tobacco/Alcohol Use

  Baseline, every 7-10 during study, within 3 days prior to surgery, and 7-14 days after surgery

Study Arms (1)

Treatment (apalutamide)

EXPERIMENTAL

Patients receive apalutamide PO on study. Patients also undergo collection of blood samples throughout the study.

Drug: Apalutamide; Procedure: Biospecimen Collection; Other: Quality-of-Life Assessment

Interventions

Apalutamide DRUG

Given PO

Also known as: ARN 509, ARN-509, ARN509, Erleada, JNJ 56021927, JNJ-56021927

Treatment (apalutamide)

Biospecimen Collection PROCEDURE

Undergo collection of blood samples

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

Treatment (apalutamide)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Treatment (apalutamide)

Eligibility Criteria

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

You may qualify if:

• Histologically confirmed organ-confined adenocarcinoma of the prostate (PCa) suitable for prostatectomy
• Gleason score =\< (4+4), however no Gleason pattern 5
• Current serum PSA =\< 20 ng/ml
• Age \> 18 years
• Karnofsky \>= 70%
• Leukocytes \>= 3,000/uL
• Absolute neutrophil count \>= 1,500/uL
• Platelets \>= 100,000/uL
• Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN) (note: in subjects with Gilbert's syndrome, if total bilirubin is \> 1.5 x ULN, measure direct and indirect bilirubin and if direct bilirubin is =\< 1.5 x ULN, subject may be eligible)
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) \< 2.5 x institutional ULN
• Creatinine \< 2 x institutional ULN
• Thyroid stimulating hormone (TSH) within the institutional normal range
• Willing to use adequate contraception (barrier method; abstinence; subject has had a vasectomy; or partner is using effective birth control or is postmenopausal) for the duration of study participation
• Ability to understand and the willingness to sign a written informed consent document

You may not qualify if:

• Prior or ongoing hormonal treatment for prostate cancer including, but not limited to orchiectomy, antiandrogens, abiraterone, ketoconazole, or estrogens, or luteinizing hormone-releasing hormone (LHRH) agonists/antagonists. Men on stable doses of 5-alpha reductase inhibitors (e.g., finasteride, dutasteride) are eligible as long as there is no planned dose change while on study
• Patients who have prostate cancer with distant metastases
• Presence of neuroendocrine differentiation in the prostate biopsies
• Serum testosterone (blood collected between 7-10 AM for men \< 45 years of age and prior to 2 PM for men \>= 45 years of age) \< 200 ng/dL
• Have a history of prior malignancies other than prostate cancer within the past 2 years, excluding non-melanoma skin cancer
• Severe or unstable angina, myocardial infarction, symptomatic congestive heart failure, arterial or venous thromboembolic events (e.g., pulmonary embolism, cerebrovascular accident including transient ischemic attacks), or clinically significant ventricular arrhythmias within 6 months prior to registration
• History of seizure or known condition that may pre-dispose to seizure (including but not limited to prior stroke, transient ischemic attack, loss of consciousness within 1 year prior to registration, brain arteriovenous malformation; or intracranial masses such as schwannomas and meningiomas that are causing edema or mass effect)
• Use of drugs known to lower the seizure threshold, including: atypical antipsychotics (e.g. clozapine, olanzapine, risperidone, ziprasidone), bupropion, lithium, meperidine, pethidine, phenothiazine antipsychotics (e.g. chlorpromazine, mesoridazine, thioridazine), and tricyclic antidepressants (e.g. amitriptyline, desipramine, doxepin, imipramine, maprotiline, mirtazapine)
• Concurrent use of drugs in category X drug interactions with apalutamide
• Participants may not be receiving any other investigational agents
• History of allergic reactions attributed to compounds of similar chemical composition of apalutamide
• Uncontrolled intermittent illnesses or medical conditions which, in the opinion of the treating physician, would make this protocol unreasonably hazardous for the patient. Such illnesses/conditions may include, but are not limited to, hypertension, ongoing or active infection, or psychiatric illness/social situations

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (5)

University of Arizona Cancer Center - Prevention Research Clinic

Tucson, Arizona, 85719, United States

Location

USC / Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

George Washington University Medical Center

Washington D.C., District of Columbia, 20037, United States

Location

Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States

Location

NCI - Center for Cancer Research

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

Prostatic Neoplasms

Interventions

apalutamide; Specimen Handling

Condition Hierarchy (Ancestors)

Genital Neoplasms, Male; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Genital Diseases, Male; Genital Diseases; Urogenital Diseases; Prostatic Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Results Point of Contact

• Title: Betsy Wertheim
• Organization: University of Arizona

betsy@arizona.edu

5207771666

Study Officials

• Juan Chipollini

  University of Arizona Cancer Center - Prevention Research Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2020
• First Posted: August 28, 2020
• Study Start: July 23, 2021
• Primary Completion: November 6, 2024
• Study Completion (Estimated): January 31, 2027
• Last Updated: May 29, 2026
• Results First Posted: August 29, 2025

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

US (5)