BMX-001 for Anal Cancer

Overseen By Erin E Rogers, BS

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Chi Lin, MD, PhD

Must be taking: 5FU, Mitomycin

Must not be taking: Corticosteroids

Disqualifiers: Breast-feeding, Active infection, Cardiovascular disease, others

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

Compensation: Varies

Number of Visits: 3

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called BMX-001 for anal cancer. The goal is to determine if BMX-001 can reduce side effects and improve outcomes when combined with the standard treatment of radiation and chemotherapy for squamous cell carcinoma of the anal canal. BMX-001 aims to protect normal tissues and enhance the destruction of cancer cells by reducing oxidative stress. People with newly diagnosed anal squamous cell carcinoma who are receiving standard chemoradiation might be suitable candidates for this trial. As a Phase 1, Phase 2 trial, this research focuses on understanding how BMX-001 works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you must be on a stable or decreasing dose of corticosteroids (a type of medication that reduces inflammation) if you are taking them.

Is there any evidence suggesting that BMX-001 is likely to be safe for humans?

Research has shown that BMX-001, when combined with radiation and chemotherapy, may reduce side effects for anal cancer patients. In one study, after six months, only one patient experienced moderate anal pain, and four reported mild stomach issues. This suggests BMX-001 is generally well-tolerated.

The study remains in its early stages, aiming to determine the safest dose of BMX-001. The treatment seeks to protect healthy tissues while helping to destroy cancer cells. By reducing damage from free radicals, BMX-001 could lessen the severe side effects often seen with standard treatments. Although safety information is still being collected, these early results offer encouragement for those considering joining the trial. ^{1 2 3 4 5}

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

BMX-001 is unique because it combines with traditional radiation therapy and chemotherapy agents like 5FU and Mitomycin to treat anal cancer, but adds a twist with its own oxidative stress-reducing properties. Unlike standard treatments that solely focus on killing cancer cells, BMX-001 works by protecting normal cells from the damage caused by radiation and chemotherapy, potentially reducing side effects. Researchers are excited about this treatment because it could enhance the effectiveness of existing therapies while making them safer and more tolerable for patients.

What evidence suggests that BMX-001 could be an effective treatment for anal cancer?

Research shows that BMX-001 is designed to protect healthy tissues and help destroy tumors by reducing stress from harmful molecules. In earlier studies, BMX-001 combined with radiation therapy extended the lives of patients with other types of cancer by an average of 6.6 months. BMX-001 also reduced inflammation and improved pain in some patients. In this trial, all participants will receive BMX-001 with standard treatment, including radiation therapy, 5FU, and Mitomycin, to evaluate its effectiveness for anal squamous cell carcinoma. While current evidence suggests potential benefits, more research is needed to confirm its effectiveness for anal cancer. ^{1 6 7 8 9}

Who Is on the Research Team?

Chi Lin, MD

Principal Investigator

University of Nebraska

Are You a Good Fit for This Trial?

This trial is for adults over 19 with newly diagnosed anal squamous cell carcinoma who need chemoradiation. They must have a good performance status, normal organ function tests, and agree to use birth control. Excluded are those breastfeeding, with active infections or other cancers needing treatment, prior pelvic radiation, certain heart conditions, uncontrolled hypertension, known allergies to the drugs involved or BMX-001.

Inclusion Criteria

I have advanced anal cancer and will be treated with chemoradiation aimed at curing it.

My blood counts meet the required levels for hemoglobin, ANC, and platelets.

Serum creatinine ≤ 1.5 mg/dl, serum SGOT and bilirubin ≤ 1.5 times upper limit of normal

Exclusion Criteria

Breast-feeding

I have a history of anal squamous cell carcinoma.

My heart's electrical cycle is longer than normal.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive BMX-001 with concurrent radiation therapy and 5FU/mitomycin chemotherapy

8 weeks

Weekly visits for radiation therapy

Follow-up

Participants are monitored for safety and effectiveness after treatment

10 months

1 month, 4 months, and 10 months post-treatment visits

Pharmacokinetics

Blood samples are drawn to evaluate the pharmacokinetics of BMX-001

36 days

Day 1, Day 8, Day 22, and Day 36

What Are the Treatments Tested in This Trial?

Interventions

• BMX-001

Trial Overview The study is testing the safety and effectiveness of BMX-001 as a radio-protector when combined with standard radiation therapy and chemotherapy (5FU/mitomycin) in treating anal cancer. Phase 1 will find the highest dose patients can take without severe side effects; Phase 2 will assess how well it reduces serious toxicity in tissues like rectum and skin.

How Is the Trial Designed?

Group I: Concurrent Radiation Therapy, 5FU, Mitomycin and BMX-001 Experimental Treatment 1 Intervention

One arm includes all enrolled patients.

Who Is Running the Clinical Trial?

Chi Lin, MD, PhD

Lead Sponsor

Trials

3

Recruited

190+

University of Nebraska

Lead Sponsor

Trials

563

Recruited

1,147,000+

BioMimetix JV, LLC

Industry Sponsor

Trials

11

Recruited

700+

Published Research Related to This Trial

Anal cancer is highly prevalent among men who have sex with men (MSM) with HIV, largely due to the high rates of human papillomavirus (HPV) infection, making early detection crucial for improving outcomes.

The review suggests implementing a coordinated screening program led by trained nurses, focusing on HPV vaccination and regular anal cytology screenings, which could significantly enhance early diagnosis and prevention of anal cancer in this high-risk population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of nurses in the prevention of anal cancer in HIV-infected men having sex with men - a focus on papillomavirus vaccination and anal cytology screening. Muñoz-Rodríguez, J., Millán-Revilla, L.[2023]

In a study analyzing archival tissue from 48 patients with localized squamous cell carcinoma of the anus (SCCA), higher protein levels of immune checkpoint markers (like PD-1) and signaling proteins (like phospho-Akt) were found in patients who experienced cancer recurrence after chemoradiation.

The findings suggest that these biomarkers could help identify patients at higher risk for recurrence and support the development of immune-based therapies to enhance treatment outcomes beyond standard chemoradiation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Differential Spatial Gene and Protein Expression Associated with Recurrence Following Chemoradiation for Localized Anal Squamous Cell Cancer. Hernandez, S., Das, P., Holliday, EB., et al.[2023]

In a study of 50 patients with anal cancer, combined chemotherapy and radiation therapy resulted in a 7-year survival rate of 58%, with better outcomes for white patients and those with favorable performance status.

Of the 46 patients evaluated for treatment response, 34 achieved a complete response, indicating that this combined modality therapy is effective, with an 80% rate of freedom from locoregional progression at 7 years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Results of combined modality therapy for patients with anal cancer (E7283). An Eastern Cooperative Oncology Group study. Martenson, JA., Lipsitz, SR., Lefkopoulou, M., et al.[2022]

Anal neoplasms are rare and currently lack standardized diagnostic and treatment protocols due to insufficient data from clinical trials.

There is a potential opportunity to develop prospective Phase II clinical trials to establish uniform guidelines for the surgical treatment of these neoplasms, which could improve patient care and outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Uncommon anal neoplasms. Billingsley, KG., Stern, LE., Lowy, AM., et al.[2010]

HIV-infected individuals are at an increased risk for anal cancer and its precursor, anal HSIL, highlighting the need for effective screening and treatment strategies for these patients.

Current treatment options for anal cancer in HIV-positive patients show variability in outcomes, and there is a pressing need for prospective, controlled trials to improve treatment efficacy and reduce morbidity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anal cancer and its precursors in HIV-positive patients: perspectives and management. Berry, JM., Palefsky, JM., Welton, ML.[2005]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03386500

Safety Study of BMX-001 (Radio-protector) in Patients With ... The use of concurrent radiation and chemotherapy with infusional 5-fluorouracil (5-FU) and mitomycin results in locoregional relapse rates of 20-32 and 5-year ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12389141/

Therapeutic Potential of BMX-001 for Preventing ... Results: Our results showed significant reductions in mechanical and cold allodynia, decreased pro-inflammatory cytokine levels, and restored ...

3. trialx.com trialx.com/clinical-trials/listings/242246/safety-study-of-bmx-001-radio-protector-in-patients-with-newly-diagnosed-anal-cancer/

Safety Study of BMX-001 (Radio-protector) in Patients With ... This allows for organ preservation in approximately 75% of patients. The use of concurrent radiation and chemotherapy with infusional 5-fluorouracil (5-FU) and ...

4. withpower.com withpower.com/trial/phase-2-neoplasms-squamous-cell-10-2017-0c17d

BMX-001 for Anal Cancer · Info for Participants This allows for organ preservation in approximately 75% of patients. The use of concurrent radiation and chemotherapy with infusional 5-fluorouracil (5-FU) and ...

5. firstwordpharma.com firstwordpharma.com/story/5802336

BioMimetix Presents Data from Phase 2 Study of BMX-001 ... In the Phase 2 study, BMX-001 in combination with radiotherapy (RT) and temozolomide (TMZ) demonstrated a 6.6 month increase in median survival.

6. redjournal.org redjournal.org/article/S0360-3016(21)01109-3/fulltext

The Phase I Results of a Phase 1/2 Trial for Patients With ... By 6 months, only 1 patient had anal pain scored 4/10 and 4 patients had G1 GI toxicity. Patients' physical, role and social functioning scores ...

7. clinicaltrial.be clinicaltrial.be/en/details/69821?per_page=20&only_recruiting=0&enrolling_by_invitation=1&active_not_recruiting=1&not_yet_recruiting=0&completed=0&only_eligible=0&only_active=0

Safety Study of BMX-001 (Radio-protector) in Patients Wit... In this Phase 1/2 study, the investigators will conduct a safety and efficacy study of the combination of BMX-001 with standard radiation ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S0360301621011093

The Phase I Results of a Phase 1/2 Trial for Patients With ... RTOG trials showed 43-87% ≥ grade (G) 3 acute toxicity. We hypothesize that BMX-001, a scavenger of O2, will decrease CRT-associated toxicity without ...

9. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2017-02375

MnSOD Mimetic BMX-001 in Treating Patients with Anal ... This phase I/II trial studies the best dose of MnSOD mimetic BMX-001 to reduce side effects in patients with anal cancer undergoing radiation therapy and ...