APG-157 for Oral Dysplasia

Overseen By Elizabeth Franzmann, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Elizabeth J Franzmann

Disqualifiers: Pregnancy, Recent surgery, Liver disease, 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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called APG-157 to determine if it can shrink tumors in individuals with oral dysplasia, a precancerous condition in the mouth. Researchers aim to understand how APG-157 affects certain markers in the body related to this condition. Participants will take APG-157 for up to 12 weeks. Individuals diagnosed with moderate to severe oral dysplasia and having a visible lesion might be suitable for this study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that APG-157 is likely to be safe for humans?

Research has shown that APG-157 is generally well-tolerated, meaning most people can use it without problems. In earlier studies, participants using APG-157 experienced positive results with few side effects. The trials indicated that this treatment did not cause major health issues. This finding reassures those considering joining the trial, suggesting that APG-157 is likely safe for people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for oral dysplasia, such as surgical removal or laser therapy, APG-157 offers a unique approach by using a new active ingredient derived from natural sources. Researchers are excited about APG-157 because it targets inflammation and abnormal cell growth in the mouth, which could potentially reduce the need for invasive procedures. This treatment's novel mechanism of action may provide a safer, non-surgical option for patients, making it a promising candidate for managing oral dysplasia.

What evidence suggests that APG-157 might be an effective treatment for oral dysplasia?

Research has shown that APG-157, the treatment under study in this trial, may help treat oral dysplasia. In one study, 6 out of 41 participants experienced a decrease in tumor size. Early results suggest that APG-157 is safe for patients and may also affect certain tumor markers. This initial evidence supports the idea that APG-157 could benefit people with oral dysplasia. ^{1 2 3 6 7}

Who Is on the Research Team?

Elizabeth Franzmann, MD

Principal Investigator

University of Miami

Are You a Good Fit for This Trial?

Adults over 18 with moderate to severe oral dysplasia or carcinoma in situ, visible lesions, and normal organ function can join. They must be able to consent, provide samples, and use contraception if needed. Excluded are those with recent fractures/surgery in the mouth area, certain allergies, liver disease, severe thrombocytopenia, pregnancy, recent cancer treatments (except some skin cancers), uncontrolled illnesses or other significant oral diseases.

Inclusion Criteria

Measurable disease - minimum lesion size of 8 x 3 mm before initial biopsy

Willing to provide blood, oral rinse, and tissue from diagnostic biopsies

Leukocytes >=3,000/microliter

Exclusion Criteria

I have had a jaw fracture in the last 8 weeks.

I have not had recent oral surgery, except for biopsies or tooth extractions, in the last 8 weeks.

Pregnant women

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive APG-157 therapy to assess reduction in tumor size and effects on tumor markers

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of lesion appearance and adverse events

16 weeks

Long-term follow-up

Participants are monitored for changes in lesion appearance and other long-term outcomes

Up to 28 months

What Are the Treatments Tested in This Trial?

Interventions

• APG-157

Trial Overview The trial is testing APG-157's ability to shrink tumors in patients with oral dysplasia. It also examines its effects on tumor markers and substances found in mouth rinses of affected individuals.

How Is the Trial Designed?

Group I: APG-157 Therapy Experimental Treatment 1 Intervention

Participants will receive APG-157 for up to 12 weeks.

Who Is Running the Clinical Trial?

Elizabeth J Franzmann

Lead Sponsor

Trials

1

Recruited

30+

Aveta Biomics, Inc.

Industry Sponsor

Trials

4

Recruited

90+

Published Research Related to This Trial

In a study of 119 oral cavity biopsy specimens, p16 immunohistochemical expression was found to be absent in 51% of cases, including a significant number of dysplastic lesions, indicating that p16 may not be a reliable biomarker for diagnosing oral epithelial dysplasia.

The research suggests that decreased p16 expression correlates with increasing severity of dysplasia, but overall, p16 immunohistochemistry does not effectively differentiate between dysplastic and nondysplastic tissues, limiting its utility in clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunohistochemical detection of p16INK4a in dysplastic lesions of the oral cavity. Bradley, KT., Budnick, SD., Logani, S.[2023]

Immunohistochemical analysis for p16INK4 protein was found to be a reliable method for identifying HPV-induced high-grade oral squamous dysplasia, with positive results in 6 out of 41 cases studied.

The presence of HPV DNA was confirmed in all 6 cases that tested positive for p16INK4, indicating a strong correlation between p16 overexpression and HPV infection, particularly with HPV-16, which may influence treatment decisions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Overexpression of p16INK4 is a reliable marker of human papillomavirus-induced oral high-grade squamous dysplasia. Cunningham, LL., Pagano, GM., Li, M., et al.[2015]

Patient advocacy groups (PAGs) play a crucial role in raising awareness about oral potentially malignant disorders (OPMD), which can lead to oral cancers, by educating both healthcare systems and patients about the importance of early diagnosis and management.

Collaboration between researchers and patient representatives enhances awareness and understanding of OPMD, which is essential for encouraging early diagnosis and effective prevention strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The 4P: Preventing Preneoplasia through Patients Partnership. Virgone, A., Badreh, S.[2021]

In a study of 60 biopsy cases, p16INK4A protein expression was found to correlate with the severity of dysplasia in oral cavity lesions, suggesting its potential as a diagnostic marker for identifying dysplastic changes.

The study revealed that p16INK4A staining was primarily located in the basal layer for mild dysplasias and in the mid to upper layers for moderate to severe dysplasias, indicating that its expression pattern could help differentiate between various grades of dysplastic lesions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Potential diagnostic use of p16INK4A, a new marker that correlates with dysplasia in oral squamoproliferative lesions. Gologan, O., Barnes, EL., Hunt, JL.[2022]

In a study of 140 oral leukoplakia lesions over 4 years, Ki-67 expression levels significantly increased from normal mucosa to dysplastic and malignant patches, indicating its potential as a reliable marker for early detection of oral cancer.

Ki-67 effectively differentiates between varying degrees of dysplasia, helping to identify mild dysplastic lesions and verrucous carcinoma from benign conditions, although it did not show significant differences in expression among different severities of oral cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Importance of Ki-67 Labeling in Oral Leukoplakia with Features of Dysplasia and Carcinomatous Transformation: An Observational Study over 4 Years. Mondal, K., Mandal, R., Sarkar, BC.[2020]

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.e18119

Preliminary results from a phase IIa trial of APG-157 in oral ... Conclusions: Preliminary pathological and clinical results suggest APG-157 is a well-tolerated and promising treatment for oral and ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05865028

A Proof of Concept Trial Investigating Safety and Efficacy ... The purpose of this study is to assess whether APG-157 can reduce the tumor size in participants with the study disease. Another purpose is to find out about ...

3. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/408175

A Proof of Concept Trial Investigating Safety and Efficacy ... The purpose of this study is to assess whether APG-157 can reduce the tumor size in participants with the study disease.

4. withpower.com withpower.com/trial/phase-2-carcinoma-in-situ-6-2023-39757

APG-157 for Oral Dysplasia Participants are monitored for safety and effectiveness after treatment ... oral squamous dysplasia, with positive results in 6 out of 41 cases studied.

5. app.trialscreen.org app.trialscreen.org/trials/phase-2-proof-concept-investigating-safety-efficacy-apg-157-oral-dysplasia-trial-nct05865028

A Phase 1 Trial of APG-157 in Oral Dysplasia Potential benefits of participating in this study include receiving an investigational treatment that may help reduce tumor size and improve outcomes for oral ...

6. cancer.gov cancer.gov/research/participate/clinical-trials/intervention/curcumin-polyphenols-containing-botanical-agent-apg-157?pn=1

Clinical Trials Using Curcumin/Polyphenols-containing ... Review the clinical trials studying curcumin/polyphenols-containing botanical agent apg-157 on this list and use the filters to refine the results by age ...

7. delta.larvol.com delta.larvol.com/Products/?ProductId=171641bc-1b73-43b0-803f-235607ac53f9

APG-157 / Aveta Biomics A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia ... - "These study results indicate that APG-157 is able to ...