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

Phase 2

Recruiting

• Conditions: Oral Cavity Dysplasia; Oropharyngeal Dysplasia; Oral Carcinoma in Situ
• Interventions: Drug: APG-157

• Registration Number: NCT05865028
• Lead Sponsor: Elizabeth J Franzmann

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-05-09
• Study First Submitted QC: 2023-05-09
• Study First Posted: 2023-05-18
• Study Start: 2023-05-24
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-02
• Last Update Posted: 2024-06-04
• Primary Completion: 2027-05-24
• Study Completion: 2028-05-24

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

1. 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.
2. Histologically proven oral cavity or oropharyngeal moderate or severe dysplasia/CIS as diagnosed by standard pathological methods and a visible lesion on oral exam.
3. Measurable disease - minimum lesion size of 8 x 3 mm before initial biopsy
4. Willing to provide blood, oral rinse and tissue from diagnostic biopsies
5. Leukocytes >=3,000/microliter
6. 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).
7. 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.
8. Able to take oral medication.
9. Able to understand and the willingness to sign a written informed consent document.

Exclusion Criteria

1. Pregnant women.
2. Subjects who have had surgery of the oral cavity, teeth, or gums within the previous 8 weeks excluding biopsies and tooth extractions.
3. Subjects who have had a fracture of the mandible or maxilla within the previous 8 weeks.
4. Inability to complete enrollment forms due to any mental status or language problems (e.g. dementia, head injury, overall illness).
5. History of prior head and neck squamous cell carcinomas (HNSCC) unless curatively treated 1 year or more prior.
6. 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.
7. Subjects with other related diseases or the oral cavity or oropharynx, as determined to be significant by the PI.
8. History of allergic reactions attributed to compounds of similar chemical composition to Curcumin (turmeric).
9. 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.
10. Severe thrombocytopenia increasing the risk of biopsy.
11. 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
APG-157 Therapy	APG-157	Participants will receive APG-157 for up to 12 weeks.

Primary Outcome Measures

Name	Time	Method
Pathologic Response Rate	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.

Secondary Outcome Measures

Name	Time	Method
Clinical Response Rate	Up to 12 weeks	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.
Change in Lesion Appearance Before and After Protocol Therapy	Baseline, 12 weeks post-intervention, Up to 28 months	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.
Number of Treatment-Related Adverse Events and Serious Adverse Events	Up to 16 weeks	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.

Trial Locations

Locations (1)

University of Miami; 🇺🇸 Miami, Florida, United States