A Proof of Concept Trial Investigating Safety and Efficacy of APG-157 in Oral Dysplasia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorElizabeth J Franzmann
About 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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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).
Trial design
Design model
Single group
Treatments tested in this trial
APG-157
DrugParticipants 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.
Treatment groups
32 Participants
are divided into 1 treatment groupGroup A: APG-157 TherapyExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
Up to 12 weeksSecondary outcomes
1
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.
Time frame
Up to 12 weeks2
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.
Time frame
Baseline, 12 weeks post-intervention, Up to 28 months3
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.
Time frame
Up to 16 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Elizabeth J Franzmann
Lead sponsor
University of Miami
Sponsor institution
Aveta Biomics, Inc.
Collaborator
Source ClinicalTrials.gov