Hydroxychloroquine for Prostate Cancer

Overseen By Bridget M Labrie

Age: 18+

Sex: Male

Trial Phase: Phase < 1

Sponsor: Lionel.D.Lewis, MD

Must not be taking: DMARDs, Calcitriol, Chloroquine

Disqualifiers: Radiation, Chemotherapy, Cardiac disease, others

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests hydroxychloroquine, a drug typically used for malaria, to determine its potential in treating prostate cancer. The primary goal is to understand how this drug affects cancer cells and assess its safety for patients planning prostate surgery. Participants must have prostate cancer that can be surgically removed and must not have undergone previous treatments like radiation or chemotherapy. The trial involves taking either the actual drug or a placebo (a harmless pill resembling the drug) before surgery. As an Early Phase 1 trial, this research aims to understand how hydroxychloroquine works in people, offering participants a chance to contribute to groundbreaking cancer treatment research.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as disease-modifying anti-rheumatic drugs (DMARDs) and any drugs that might affect autophagy, like calcitriol and chloroquine. If you are taking medications that can cause a heart rhythm problem called torsades de pointes, you must stop them for a period longer than six times their half-life before joining the study.

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

Research has shown that hydroxychloroquine (HCQ) is generally well-tolerated by patients in early studies. It has been tested in lab models and trials to slow tumor growth, showing potential as a cancer treatment. This drug blocks a process called autophagy, which cancer cells use to survive.

Previous studies on hydroxychloroquine have not identified major safety issues. However, like any medication, it can cause side effects, such as mild nausea or dizziness. This trial is in its early stages, so scientists are still gathering detailed safety information.

For those considering joining this trial, it may be reassuring to know that hydroxychloroquine has been studied for other conditions and is generally considered safe. However, always consult healthcare professionals before making a decision. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for prostate cancer currently involve hormone therapy, chemotherapy, or radiation. But hydroxychloroquine works differently, targeting cancer cells' survival mechanisms by interfering with autophagy, a process cells use to break down and recycle components. Researchers are excited because hydroxychloroquine could potentially enhance the effectiveness of existing therapies by making cancer cells more susceptible to treatment. Plus, it's an oral medication, which might offer a more convenient option for patients compared to some traditional therapies.

What evidence suggests that hydroxychloroquine might be an effective treatment for prostate cancer?

Research suggests that hydroxychloroquine (HCQ), which participants in this trial may receive, might help treat prostate cancer. HCQ has been found to increase the production of a protein called PAR-4, which can slow tumor growth. Studies also indicate that HCQ affects certain processes involved in cancer development, offering a hopeful approach to managing the disease. Additionally, HCQ blocks a process called autophagy, which cancer cells use to survive, making it a potential anticancer treatment. Overall, early results are promising, but more research is needed to confirm its effectiveness specifically for prostate cancer. ^{1 2 4 5 6}

Who Is on the Research Team?

Lionel Lewis, MB BCh., MD

Principal Investigator

Dartmouth-Hitchcock Medical Center

Are You a Good Fit for This Trial?

Men with resectable prostate cancer, confirmed by pathology as adenocarcinoma Gleason score 6 or higher, who are fit for radical prostatectomy can join. They need enough biopsy tissue for study analysis and must have normal blood counts, liver and kidney function. Participants must understand the investigational nature of the study.

Inclusion Criteria

My prostate cancer has a Gleason score of 6 or higher.

I am medically fit for major prostate surgery.

My prostate cancer can be surgically removed and I am planning to have surgery.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral hydroxychloroquine or placebo for up to 28 days prior to surgical resection

4 weeks

Daily oral administration

Surgery

Participants undergo radical prostatectomy to assess the effects of hydroxychloroquine on prostate cancer tissue

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment and surgery

4 weeks

Post-surgery visit

What Are the Treatments Tested in This Trial?

Interventions

• Hydroxychloroquine

Trial Overview

The trial is testing if hydroxychloroquine affects autophagy markers in prostate cancer tissues compared to a placebo. Patients will take the drug before surgery to remove their prostate. The study also looks at drug levels in blood and tissue, side effects, and links between treatment response and tumor genetics.

How Is the Trial Designed?

Group I: Hydroxychloroquine Experimental Treatment 1 Intervention

Loading dose 400mg three times a day then 400mg twice daily for up to 28 days prior to surgical resection, taken orally

Group II: Placebo Placebo Group 1 Intervention

Loading dose three times a day then twice daily for up to 28 days prior to surgical resection, taken orally

Who Is Running the Clinical Trial?

Lionel.D.Lewis, MD

Lead Sponsor

Trials

1

Recruited

20+

Dartmouth Cancer Center

Collaborator

Trials

3

Recruited

260+

Citations

1.

ascopubs.org

ascopubs.org/doi/10.1200/JCO.2025.43.5_suppl.233

Hydroxychloroquine effects on tumor suppressor PAR-4 ...

Hydroxychloroquine (HCQ) has been identified to be a potent inducer of PAR-4 secretion and downstream tumor inhibition in preclinical models and Phase I trials.

2.

nature.com

nature.com/articles/s41598-025-04267-y

Hydroxychloroquine modulates the progression of ...

Our findings suggest that HCQ modulates BPH progression by targeting STAT3/FOXO1/TRAIL and EGFR/ERK/AR pathways, offering a promising therapeutic strategy for ...

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04011410

Hydroxychloroquine to Increase Tumor Suppressor PAR-4 ...

Potential benefits of Hydroxychloroquine include delayed disease progression and delayed initiation of ADT, lessening morbidity and increasing quality of life ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5718030/

chloroquine and hydroxychloroquine as anti-cancer agents

The therapy was reported to be relatively safe, and the overall response rate was 40%, with an 84% disease control rate [54]. However, this was ...

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S0009279723004179

Hydroxychloroquine: Key therapeutic advances and ...

Hydroxychloroquine (HCQ) is a promising anticancer agent in both monotherapy and combined therapy. HCQ may be able to fight cancer through autophagy inhibition.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC5655212/

Hydroxychloroquine and risk of cancer in patients with primary ...

A recent meta-analysis summarized data from 14 studies found that patients with pSS had a pooled relative risk of 1.53 for overall cancer and 13.76 for non- ...