Erdafitinib for Castration-Resistant Prostate Cancer

Age: 18+

Sex: Male

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must be taking: AR-targeting agents

Disqualifiers: Hypertension, Eye conditions, Cardiovascular, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 21 days per cycle, up to 5 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests erdafitinib, a drug designed to stop prostate cancer that grows despite treatments blocking male hormones (castration-resistant prostate cancer). The researchers aim to determine if erdafitinib can control the disease by blocking enzymes that promote cancer cell growth. Blood, tissue, and bone marrow samples will be collected to study DNA changes and identify cancer markers. Men with prostate cancer that has spread to the bones and who have tried other hormone-blocking treatments might be suitable candidates for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it does mention that you should not have received any other investigational agent or participated in another clinical study within 30 days prior to starting this trial.

Is there any evidence suggesting that erdafitinib is likely to be safe for humans?

Research has shown that erdafitinib has been tested in patients with various types of cancer, including prostate cancer that doesn't respond to hormone treatment. The data suggests that erdafitinib is usually well-tolerated, but some side effects require consideration. For instance, some patients experienced a return or worsening of a vision problem called central serous retinopathy (CSR) after restarting erdafitinib following a break in treatment. This indicates that while the treatment can be effective, it may carry certain risks.

Since this trial is in phase 2, erdafitinib has already demonstrated some safety in earlier studies, but researchers continue to gather more detailed safety information. Participants should discuss potential risks and benefits with their healthcare provider before joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for prostate cancer?

Unlike the standard treatments for castration-resistant prostate cancer, which often include hormone therapies and chemotherapy, erdafitinib targets a specific pathway involved in cancer growth. Erdafitinib is unique because it inhibits the fibroblast growth factor receptor (FGFR), which is a new approach compared to traditional treatments. Researchers are excited about this treatment because targeting FGFR could potentially stop the cancer from progressing, offering a new hope for patients who have limited options. Additionally, erdafitinib is taken orally, which can be more convenient and less invasive than some other treatments.

What evidence suggests that erdafitinib might be an effective treatment for castration-resistant prostate cancer?

Studies have shown that erdafitinib can help manage castration-resistant prostate cancer, a type of cancer that continues to grow despite treatments that lower testosterone levels. This drug blocks certain enzymes crucial for tumor growth. Research indicates that this type of prostate cancer can adapt to hormone treatments, but erdafitinib targets a different pathway, offering a new approach. Findings suggest that by targeting the fibroblast growth factor pathway, erdafitinib could effectively slow or stop tumor growth. While more data is needed, early research supports its potential to control this aggressive form of cancer. Participants in this trial will receive erdafitinib as part of the study treatment. ^{3 6 7 8 9}

Who Is on the Research Team?

Paul Corn

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

Men aged 18+ with castration-resistant prostate cancer and bone metastases are eligible. They must have had prior treatments like hormone therapy or chemotherapy, but not recent major surgery or radiation. Participants need to maintain low testosterone levels, have adequate organ function, be able to swallow pills, and agree to use contraception.

Inclusion Criteria

You have a sufficient amount of a type of white blood cell called neutrophils.

Your AST or ALT levels in your blood are within a certain range.

Your bilirubin levels in the blood are within a certain range, unless you have a condition called Gilbert's disease.

Exclusion Criteria

I haven't been part of any drug trials or received experimental treatments in the last 30 days.

You have a current hepatitis B or C infection.

I am still experiencing side effects from previous cancer treatments.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive erdafitinib orally once daily on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

21 days per cycle, up to 5 years

Regular visits for treatment and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment completion. Follow-up occurs at 30 days, every 16 weeks for 1 year, and then every 6 months thereafter.

Up to 5 years

Follow-up visits at specified intervals

What Are the Treatments Tested in This Trial?

Interventions

• Erdafitinib

Trial Overview The trial is testing Erdafitinib's effectiveness in treating advanced prostate cancer that resists standard treatment. It involves taking the drug orally and monitoring its impact on tumor growth by blocking enzymes needed for cell growth.

How Is the Trial Designed?

Group I: Treatment (erdafitinib, biospecimen collection) Experimental Treatment 3 Interventions

Patients receive erdafitinib PO QD on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients may also undergo collection of blood and bone marrow via biopsy and aspirates.

Erdafitinib is already approved in United States, European Union for the following indications:

Approved in United States as Balversa for:

• Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations

Approved in European Union as Balversa for:

• Locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Published Research Related to This Trial

Erlotinib, an EGFR inhibitor, showed reduced effectiveness in treating castration-resistant prostate cancer, particularly when the EGFR/Her2 ratio was low, which was influenced by chronic treatment with bicalutamide.

The study suggests that erlotinib may be more effective in hormone-naive prostate cancer patients rather than those who have undergone hormonal treatment, as low levels of Her2 and a higher EGFR/Her2 ratio were associated with better drug efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of EGFR tyrosine kinase inhibitor erlotinib in prostate cancer cells in vitro. Festuccia, C., Gravina, GL., Biordi, L., et al.[2018]

In a phase II study involving 30 patients with advanced prostate cancer, erlotinib showed moderate toxicity but resulted in clinical benefit for 40% of participants, indicating some effectiveness in improving patient outcomes.

While no patients experienced a decrease in prostate-specific antigen (PSA) levels, 14% had stabilization of PSA, and 10 patients showed a significant increase in PSA-doubling time, suggesting potential for further investigation in earlier stages of prostate cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Results from a monocentric phase II trial of erlotinib in patients with metastatic prostate cancer. Gravis, G., Bladou, F., Salem, N., et al.[2020]

In a phase 2 study involving 51 men with castration-resistant prostate cancer (CRPC), gefitinib showed minimal clinical efficacy, with only 2% of patients achieving a significant reduction in prostate-specific antigen (PSA) levels.

The treatment was associated with notable side effects, leading to dose reductions in 13 patients and withdrawal in 9 due to adverse events, indicating that while some patients maintained stable performance status, the overall response to gefitinib was limited.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An open-label, single-arm phase two trial of gefitinib in patients with advanced or metastatic castration-resistant prostate cancer. Pezaro, C., Rosenthal, MA., Gurney, H., et al.[2018]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04754425?intr=BALVERSA&aggFilters=studyType:int&viewType=Table&rank=2

Erdafitinib for the Treatment of Patients With Castration ... Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37796107/

Targeting the fibroblast growth factor pathway in molecular ... However, castration-resistant prostate cancer (CRPC) tumors can resist AR signaling inhibitors through AR amplification and AR splice variants ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10851871/

Targeting the Fibroblast Growth Factor Pathway in ... However, castration-resistant prostate cancer (CRPC) tumors can resist AR signaling inhibitors through AR amplification and AR splice variants in AR-positive ...

4. frontiersin.org frontiersin.org/journals/oncology/articles/10.3389/fonc.2025.1571434/full

Meta-analysis on the safety and efficacy of Erdafitinib in ... ... outcomes. This evidence substantiates further evaluation of ... Castration-Resistant Prostate Cancer: A Systematic Review and Meta-Analysis.

5. mdanderson.org mdanderson.org/patients-family/diagnosis-treatment/clinical-trials/clinical-trials-index/clinical-trials-detail.ID2020-0953.html

A Study of Erdafitinib in Castration-Resistant Prostate ... Giving erdafitinib may help control disease in patients with castration-resistant prostate cancer. In addition, studying samples of blood, tissue, plasma, and ...

6. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT04754425

Erdafitinib for the Treatment of Patients With Castration ... Giving erdafitinib may help control disease in patients with castration-resistant prostate cancer. In addition, studying samples of blood, tissue, plasma, and ...

7. balversahcp.com balversahcp.com/clinical-information/safety

Safety | BALVERSA® (erdafitinib) HCP Of the 24 patients who restarted BALVERSA® after dose interruption with or without dose reduction, 67% had recurrence and/or worsening of CSR after restarting.

8. app.trialscreen.org app.trialscreen.org/trials/phase-2-castration-resistant-prostate-carcinoma-erdafitinib-treatment-patients-trial-nct04754425

Erdafitinib for the Treatment of Patients With Castration ... This study evaluates the efficacy, objective response rate, progression-free survival, and safety profile of erdafitinib treatment. Patients ...

9. balversa.com balversa.com/

BALVERSA® (erdafitinib): Homepage The official BALVERSA® patient site. See Full Safety and Prescribing Information.