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

Primary Purpose

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

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

APG-157

About this trial

This is an interventional treatment trial for Head and Neck Cancer focused on measuring Neoadjuvant, Induction Therapy, Oral Cancer, Oropharyngeal Cancer

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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

Sites / Locations

VAGLAHS, West Los AngelesRecruiting
University of Miami Sylvester Comprehensive Cancer CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

APG-157

Arm Description

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

Outcomes

Primary Outcome Measures

Imaging to assess drug's ability to impact tumor size

Change in Tumor Size from Baseline to end of dosing using MRI with or without contrast and PET/CT imaging.

Secondary Outcome Measures

Biopsy

Change in Immunohistochemistry profile (including the levels of tumor infiltrating lymphocytes TILs) of tumor biopsy from baseline to end of dosing.

Saliva Profile

Change in cytokine levels (IL-1β , TNF-α, IL-8) and oral microbiome (phyla and genus level changes in microbial composition using 16s RNA sequencing) in saliva from baseline to end of dosing.

Cell-free RNA analyses of saliva and blood

Change in cell-free RNA biomarkers in blood and saliva from baseline to end of dosing.

Full Information

NCT ID

NCT05312710

First Posted

March 16, 2022

Last Updated

August 1, 2023

Sponsor

Aveta Biomics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05312710

Brief Title

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

Official Title

Phase 2 Study to Evaluate the Safety and Efficacy of APG-157 as Neoadjuvant/Induction Therapy for Patients With Head and Neck Squamous Cell Cancer (HNSCC) of the Oral Cavity and/or Oropharynx

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Recruiting

Study Start Date

April 22, 2022 (Actual)

Primary Completion Date

July 2024 (Anticipated)

Study Completion Date

July 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Aveta Biomics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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: 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. 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. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

Keywords

Neoadjuvant, Induction Therapy, Oral Cancer, Oropharyngeal Cancer

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

24 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

APG-157

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

APG-157

Intervention Description

Treatment

Primary Outcome Measure Information:

Title

Imaging to assess drug's ability to impact tumor size

Description

Change in Tumor Size from Baseline to end of dosing using MRI with or without contrast and PET/CT imaging.

Time Frame

Baseline to end of dosing is four weeks (extendable up to 6 weeks) of APG-157 dosing. Baseline is at time of diagnosis. End of dosing is day before surgery or start of other definitive therapy and/or radiation

Secondary Outcome Measure Information:

Title

Biopsy

Description

Change in Immunohistochemistry profile (including the levels of tumor infiltrating lymphocytes TILs) of tumor biopsy from baseline to end of dosing.

Time Frame

Title

Saliva Profile

Description

Change in cytokine levels (IL-1β , TNF-α, IL-8) and oral microbiome (phyla and genus level changes in microbial composition using 16s RNA sequencing) in saliva from baseline to end of dosing.

Time Frame

Title

Cell-free RNA analyses of saliva and blood

Description

Change in cell-free RNA biomarkers in blood and saliva from baseline to end of dosing.

Time Frame

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Marilene B Wang, MD

Phone

310 268-3748

marilene.wang@va.gov

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Marilene B Wang, MD

Organizational Affiliation

VA Los Angeles/UCLA

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Elizabeth Franzmann, MD

Organizational Affiliation

University of Miami Sylvester Comprehensive Cancer Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

VAGLAHS, West Los Angeles

City

Los Angeles

State/Province

California

ZIP/Postal Code

90073

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Marilene B Wang, MD

Phone

310-268-3748

marilene.wang@va.gov

Facility Name

University of Miami Sylvester Comprehensive Cancer Center

City

Miami

State/Province

Florida

ZIP/Postal Code

33136

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Elizabeth Franzmann, MD

Phone

305-243-5955

EFranzmann@med.miami.edu

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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