Safety and Efficacy of APG-157 in Head and Neck Cancer

NCT ID: NCT05312710

Last Updated: 2026-01-16

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-04-22
• Study Completion Date: 2026-12-31

Brief Summary

The purpose of this clinical research study is to study safety and efficacy of orally administered APG-157 as the neoadjuvant/induction therapy in newly diagnosed, locally advanced patients with Head & Neck Cancer of oral cavity and/or oropharynx.

The study hypothesis is that neoadjuvant use of APG-157 will reduce the tumor burden prior to any definitive therapy to improve the outcomes over current standard of care.

Detailed Description

The patient will receive neoadjuvant therapy (APG-157) during the period between initial diagnosis and time of definitive treatment. APG-157 is an orally administered drug in a form of pastille (soft lozenge) taken three times a day. It dissolves in the mouth. After the neoadjuvant treatment, the patient undergoes surgery or any other definitive therapy and/or postoperative radiotherapy as determined by the patient's doctor. Duration of treatment is four weeks that may be extended up to six weeks by mutual consent of the patients and the investigators.

Objectives:

1. To conduct Phase 2A to determine how tumor size, tumor tissue biomarkers, and the cancer stem cell markers, in head and neck squamous cell cancer patients are affected by the administration APG-157 pastilles using imaging and other clinical measurements.

2. To determine the degrees of response of each patient to APG-157 (considering patient's diagnosis/staging and local treatment) using proposed primary, secondary and exploratory endpoints.

3. The results from this study will be used to finalize the design of subsequent Phase 2B study (single arm for specific patient population and local treatment) to demonstrate statistically significant efficacy outcomes.

Conditions

Head and Neck Cancer; Squamous Cell Carcinoma of Oral Cavity; Squamous Cell Carcinoma of the Oropharynx

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

APG-157

Two pastilles (100 mg) taken three times a day (i.e. before meal time).

Group Type: EXPERIMENTAL

APG-157

Intervention Type: DRUG

Treatment

Interventions

APG-157

Treatment

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

A. Biopsy proven oral cavity or oropharyngeal Squamous Cell Carcinoma.

B. Newly diagnosed, treatment naive Stage I, Stage II, Stage III or Stage IV HNSCC patients. Staging is done according to the International Union Against Cancer's (UICC) classification system for oral cancer. Acceptable TNM staging is T1-4, N0-2, M0.

C. Patients who are scheduled to receive the following therapy after APG-157 treatment.

1. Local Therapy with Curative Intent Surgery alone or surgery followed by radiation.

2. Therapy with Palliative Intent Radiation alone. Radiation with concurrent radiosensitizing chemotherapeutic agents only using QUAD-shot protocol. Radiosensitizing chemotherapeutic agents are limited to carboplatin or cetuximab.

3. Patients who refuse surgery or are unfit for any local therapy.

Exclusion Criteria

A. Patients whose definitive, local treatment is available in less than four weeks from initial diagnosis. For example, some patients who are scheduled to receive chemo-radiation therapy as the local therapy with curative intent.

B. Pregnant women.

C. Prior Chemotherapy or radiation therapy within the last 8 weeks.

D. Patients with recurrent or metastatic cancer.

E. Tooth abscesses.

F. Bleeding gums or cracked teeth.

G. Patients who have had surgery of the oral cavity, teeth, or gums within the previous 8 weeks.

H. Patients who have had a fracture of the mandible or maxilla within the previous 8 weeks.

I. Inability to complete enrollment forms due to any mental status or language problems (e.g. dementia, head injury, overall illness).

J. Patients with other related diseases or the oral cavity or oropharynx, as determined to be significant by the PI.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Aveta Biomics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Marilene B Wang, MD

Role: PRINCIPAL_INVESTIGATOR

VA Los Angeles/UCLA

Elizabeth Franzmann, MD

Role: PRINCIPAL_INVESTIGATOR

University of Miami Sylvester Comprehensive Cancer Center

Locations

VAGLAHS, West Los Angeles

Los Angeles, California, United States

University of Miami Sylvester Comprehensive Cancer Center

Miami, Florida, United States

Countries

United States

Other Identifiers

AVTA 20-01

Identifier Type: -

Identifier Source: org_study_id