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

Trial Summary

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.

What makes the drug APG-157 unique for treating oral dysplasia?

The research provided does not contain specific information about APG-157, so I cannot determine what makes it unique for treating oral dysplasia. ^{1 2 3 4 5}

What is the purpose of this trial?

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 the effects of APG-157 on certain tumor markers and oral rinses in participants with the study disease.

Research Team

Elizabeth Franzmann, MD

Principal Investigator

University of Miami

Eligibility Criteria

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

I have a confirmed severe gum or throat condition with a visible sore.

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

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

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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]

References

1. India 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. [2020]

2. Switzerland pubmed.ncbi.nlm.nih.gov

The 4P: Preventing Preneoplasia through Patients Partnership. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Potential diagnostic use of p16INK4A, a new marker that correlates with dysplasia in oral squamoproliferative lesions. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Immunohistochemical detection of p16INK4a in dysplastic lesions of the oral cavity. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Overexpression of p16INK4 is a reliable marker of human papillomavirus-induced oral high-grade squamous dysplasia. [2015]