Oropharyngeal DysplasiaPhase 2NCT05865028
A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia
A Phase 2 study testing APG-157 in people with Oropharyngeal Dysplasia and Oral Cavity Dysplasia, run by Elizabeth J Franzmann at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oropharyngeal Dysplasia, Oral Cavity Dysplasia, Oral Carcinoma in Situ
- What is being tested?
- APG-157
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 24 May 2023to 24 May 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
- Absolute neutrophil count >= 1,000/microliter, Platelets >= 100,000/microliter, Total bilirubin =< 1.5 x institutional upper limit of normal (ULN), aspartate aminotransferase (AST), serum glutamic-oxaloacetic transaminase (SGOT), alanine aminotransferase (ALT) and serum glutamic pyruvic transaminase (SGPT) =< 1.5 x institutional upper limit of normal (ULN). Exceptions to this Inclusion Criteria can be made if labs are deemed not clinically significant by the principal investigator (PI).
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- 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.
Who cannot join
- 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.
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- 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).
- Active or chronic liver disease, evidence of hepatitis (infectious or autoimmune), cirrhosis or portal hypertension. Exceptions to this Exclusion Criteria can be made if patient status is deemed not clinically significant by the PI.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Miami
Miami, Florida, 33136
Elizabeth J Franzmann, MD, +1 (305) 243-5955, efranzman@med.miami.edu
Elizabeth J Franzmann, MD
About this study
Official title: Phase II A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia
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.
- Being tested
- APG-157
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APG-157Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| APG-157 Therapy | Experimental | Participants will receive APG-157 for up to 12 weeks. Receives: Drug: APG-157 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 12 weeks
Secondary outcomes (3)
Clinical Response Rate
The clinical response rate among participants receiving study treatment will be assessed. The clinical response rate is defined as the percentage of participants with complete response (CR) and/or partial response (PR) 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.
Measured: Up to 12 weeks
Change in Lesion Appearance Before and After Protocol Therapy
Change in lesion appearance will be assessed using criteria consistent with Response Evaluation Criteria in Solid Tumors (RECIST) v.1.1 criteria, found at time of oral exam per physician discretion.
Measured: Baseline, 12 weeks post-intervention, Up to 28 months
Number of Treatment-Related Adverse Events and Serious Adverse Events
The number of treatment-related adverse events (AEs) and serious adverse events (SAEs) in participants receiving protocol therapy will be assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5, per physician discretion.
Measured: Up to 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2023
- Study start24 May 2023
- Primary completion24 May 2027 (estimated)
- Study completion24 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oropharyngeal DysplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Elizabeth J Franzmann
- Sponsor type
- University, hospital or other
- Collaborators
- Aveta Biomics, Inc.
- Responsible party
- Sponsor-investigator, Elizabeth J Franzmann, University of Miami
- Investigators
- Elizabeth J Franzmann, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05865028
- Sponsor's protocol ID
- 20221112
- Other IDs
- NCI-2023-04230
- Status last verified
- Dec 2025
- First submitted
- 9 May 2023
- Last updated
- 14 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia
- ClinicalTrials.gov ID
- NCT05865028
- Acronym / sponsor ID
- 20221112
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Elizabeth J FranzmannUniversity, hospital or other
- Collaborators
- Aveta Biomics, Inc.
- Study start
- 24 May 2023 (actual)
- Primary completion
- 24 May 2027 (estimated)
- Study completion
- 24 May 2028 (estimated)
- First posted
- 18 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| APG-157 Therapy | Experimental | Drug: APG-157Participants will receive APG-157 for up to 12 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| APG-157Participants 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. | Drug | APG-157 Therapy |
Primary outcome
Pathologic Response Rate
Time frame: Up to 12 weeks
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.
3 secondary outcomes
Clinical Response Rate
Time frame: Up to 12 weeks
Change in Lesion Appearance Before and After Protocol Therapy
Time frame: Baseline, 12 weeks post-intervention, Up to 28 months
Number of Treatment-Related Adverse Events and Serious Adverse Events
Time frame: Up to 16 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Elizabeth J Franzmann, MD principal investigator
University of Miami
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing APG-157 in people with Oropharyngeal Dysplasia and Oral Cavity Dysplasia, run by Elizabeth J Franzmann at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Elizabeth J Franzmann, MD (305-243-5955, efranzman@med.miami.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pathologic Response Rate (time frame: Up to 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2026.