A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia

NCT05865028 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 20221112NCI-2023-04230
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Oropharyngeal Dysplasia; Oral Cavity Dysplasia; Oral Carcinoma in Situ

Outcome Measures

Primary Outcomes (1)

• Pathologic Response Rate

  The pathologic response rate among participants receiving study treatment will be assessed. The pathologic response rate is defined as the percentage of participants with mild or no dysplasia after receiving study therapy. Response will be assessed on the basis of clinical, radiologic, molecular and pathologic criteria consistent with Response Evaluation Criteria in Solid Tumors (RECIST) v.1.1 criteria, per physician discretion.

  Up to 12 weeks

Secondary Outcomes (3)

• Clinical Response Rate

  Up to 12 weeks

• Change in Lesion Appearance Before and After Protocol Therapy

  Baseline, 12 weeks post-intervention, Up to 28 months

• Number of Treatment-Related Adverse Events and Serious Adverse Events

  Up to 16 weeks

Study Arms (1)

APG-157 Therapy

EXPERIMENTAL

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

Drug: APG-157

Interventions

APG-157 DRUG

Participants will take 200mg (2 x 100mg pastilles) of APG-157 therapy orally (PO) three times daily during each 4-week cycle for up to three cycles. Cycle three is optional.

APG-157 Therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients age \> 18 years with biopsy-proven moderate to severe oral dysplasia or carcinoma in situ (CIS) and a visible lesion. Sites include all oral cavity as well as oropharyngeal dysplasia that is accessible in the outpatient clinic.
• Histologically proven oral cavity or oropharyngeal moderate or severe dysplasia/CIS as diagnosed by standard pathological methods and a visible lesion on oral exam.
• 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
• Willing to use adequate contraception, subject or partner has had a vasectomy or partner is using effective birth control or is post-menopausal for the duration of the study.
• Able to take oral medication.
• Able to understand and the willingness to sign a written informed consent document.

You may not qualify if:

• Pregnant women.
• Subjects who have had surgery of the oral cavity, teeth, or gums within the previous 8 weeks excluding biopsies and tooth extractions.
• Subjects who have had a fracture of the mandible or maxilla within the previous 8 weeks.
• Inability to complete enrollment forms due to any mental status or language problems (e.g. dementia, head injury, overall illness).
• History of prior head and neck squamous cell carcinomas (HNSCC) unless curatively treated 1 year or more prior.
• Use of chemotherapy and/or radiation for any malignancy (excluding nonmelanoma skin cancer and cancer confined to organs with removal as only treatment) in the past 2 years.
• Subjects with other related diseases or the oral cavity or oropharynx, as determined to be significant by the PI.
• History of allergic reactions attributed to compounds of similar chemical composition to Curcumin (turmeric).
• Severe thrombocytopenia increasing the risk of biopsy.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, uncontrolled congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

Sponsors & Collaborators

• Elizabeth J Franzmann: lead
• Aveta Biomics, Inc.: collaborator

Study Sites (1)

University of Miami

Miami, Florida, 33136, United States

RECRUITING

Study Officials

• Elizabeth J Franzmann, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Elizabeth J Franzmann, MD

CONTACT

305-243-5955; efranzman@med.miami.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 9, 2023
• First Posted: May 18, 2023
• Study Start: May 24, 2023
• Primary Completion (Estimated): May 24, 2027
• Study Completion (Estimated): May 24, 2028
• Last Updated: January 14, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)