Proton Therapy for Anal Cancer

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Jordan Kharofa

Disqualifiers: Life expectancy < 3 months

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

Compensation: Varies

Number of Visits: Unknown

Duration: 3 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether proton therapy, a type of radiation treatment, can reduce the side effects of standard therapy for anal cancer. It aims to lower radiation exposure to healthy areas while effectively targeting cancer cells. Participants will receive a combination of proton therapy and chemotherapy (cytotoxic therapy). Suitable candidates have specific types of anal cancer and are in moderate to advanced stages of the disease. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that proton therapy might be safer than traditional radiation for treating anal cancer. Studies have found that patients experience fewer side effects with proton therapy because it targets cancer more precisely, reducing the impact on healthy tissues.

The chemotherapy treatment involves two drugs: 5-Fluorouracil (5-FU) and Mitomycin. These drugs are commonly used for anal cancer and are generally well-tolerated. Research has indicated that patients treated with these drugs often have good survival rates, demonstrating their safety.

In summary, studies have shown that both proton therapy and the chemotherapy drugs used are safe. Always consult medical professionals for advice specific to individual health needs. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Proton therapy for anal cancer is unique because it uses pencil beam proton radiotherapy, which is a more precise form of radiation therapy. Unlike traditional radiation treatments that use X-rays, proton therapy delivers radiation directly to the tumor with minimal damage to surrounding healthy tissues. Researchers are excited because this precision could lead to fewer side effects and improved quality of life for patients, while still effectively targeting the cancer. Additionally, when combined with standard chemotherapy drugs like 5-FU and Mitomycin, it may enhance the overall effectiveness of the treatment.

What evidence suggests that Proton Therapy and Chemotherapy could be effective for anal cancer?

Research has shown that proton therapy, when combined with chemotherapy, could effectively treat anal cancer. In this trial, participants will receive both proton therapy and chemotherapy. Studies have found that proton therapy better protects healthy areas, such as the small bowel and bladder, from radiation compared to traditional X-ray treatments. This protection might lead to fewer side effects, benefiting patients. Early results have also shown high rates of complete clinical response (cCR) and two-year disease-free survival. Overall, proton therapy may offer an advantage by targeting cancer while sparing normal tissues. ^{6 7 8 9 10}

Who Is on the Research Team?

Jordan Kharofa, MD

Principal Investigator

University of Cincinnati

Are You a Good Fit for This Trial?

This trial is for individuals with a specific type of anal cancer (squamous or basaloid carcinoma) that's at a certain stage (T2-4, any N category). Participants must be relatively active and able to care for themselves (Karnofsky Performance Status >70%). Those expected to live less than 3 months cannot join.

Inclusion Criteria

Histologically documented squamous or basaloid carcinoma of the anal canal

Karnofsky Performance Status >70%

Your cancer has spread beyond its original location (Stage T2-4) and may have spread to nearby lymph nodes (any N category).

Exclusion Criteria

Doctors do not expect you to live more than 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive standard chemoradiation using 5-FU, Mitomycin, with pencil beam proton radiotherapy

3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

60 months

Quality of Life Assessment

Utilization of the Patient Reported Outcomes- Common Toxicity Criteria for Adverse Events at pretreatment and up to 12 months

12 months

What Are the Treatments Tested in This Trial?

Interventions

• Chemotherapy
• Proton therapy

Trial Overview The study is testing if Proton Therapy can reduce the radiation received by healthy tissues around anal cancer compared to standard therapy. The goal is to see if this method lowers side effects while treating the cancer effectively.

How Is the Trial Designed?

Group I: Proton Therapy and Chemotherapy Experimental Treatment 2 Interventions

Standard chemoradiation using 5-FU, Mitomycin, with pencil beam proton radiotherapy

Chemotherapy is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:

Approved in European Union as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Approved in United States as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Approved in Canada as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Approved in Japan as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Approved in China as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Approved in Switzerland as Chemotherapy for:

• Breast cancer
• Metastatic breast cancer
• Various other cancers

Who Is Running the Clinical Trial?

Jordan Kharofa

Lead Sponsor

Trials

2

Recruited

10+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11433675/

Evaluating the Effectiveness of Proton Beam Therapy ... Proton therapy and mixed plans provided better sparing of the small bowel compared to X-rays, with bladder risk being at 1% for proton therapy, ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S2949819825000184

Review Innovative radiotherapy approaches Conclusively, several studies for anal cancer and other pelvic tumors have shown a dosimetric benefit of proton therapy compared with IMRT ...

3. jgo.amegroups.org jgo.amegroups.org/article/view/28524/html

Proton beam radiotherapy for anal and rectal cancers - Vaios There were 2 deaths on study which were unrelated to radiation. At a median follow-up of 26 months, the cCR rate and 2-year disease-free survival rate were 88% ...

4. tandfonline.com tandfonline.com/doi/full/10.3109/0284186X.2014.1002570

Pencil-beam scanning proton therapy for anal cancer Results. Compared to IMRT, PBT reduced low dose radiation (≤ 30 Gy) to the small bowel, total pelvic bone marrow, external genitalia, femoral heads, ...

5. advancesradonc.com advancesradonc.com/article/S2452-1094(21)00102-0/fulltext

Multi-institutional Comparison of Intensity Modulated ... Scanning proton beam therapy reduces normal tissue exposure in pelvic radiotherapy for anal cancer ... Comparative Effectiveness of Proton vs ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8842324/

Outcomes and Toxicity of 428 Patients Treated at a Single ... Two- and 5-year freedom from locoregional failure, freedom from colostomy failure, and overall survival were 86.5% and 81.2%, respectively, 90.0 ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/19203845/

Outcomes of chemoradiotherapy with 5-Fluorouracil and ... Three-year overall survival was 83.6% (95% CI = 68.2-100) and 91.7% (95% CI = 77.3-100) in the IC and ID groups, respectively. In addition ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S294981982500010X

Review Optimising chemoradiotherapy in anal cancer It is reassuring that the standard 5-FU/MMC arms in all three trials reported very similar 5-year OS at 78%, 79%, and 71% in the RTOG 98-11, ACT ...

9. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/1028636

Fluorouracil, Mitomycin, and Radiotherapy vs ... The 3- and 5-year overall survival rates were 84% (95% CI, 78%-88%) and 75% (95% CI, 67%-81%), respectively, in the mitomycin-based group and 76 ...

10. ascopubs.org ascopubs.org/doi/10.1200/JCO.2022.40.4_suppl.003

Are two too many when it comes to the treatment of anal ... Conclusions: Our analysis showed that the patterns of failure and the risk of loco-regional and distant failures were similar between patients ...