A Study of BB-401 in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety, tolerability and efficacy of intratumoral injections with an Epidermal Growth Factor Receptor (EGFR) AntiSense DNA (BB-401) in patients with metastatic/recurrent HNSCC.

Official Title
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A Phase 2 Trial of Intratumoral EGFR Antisense DNA (BB-401) in Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck (HNSCC) Who Have Failed All Available Standard Therapies

Conditions
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Head and Neck Squamous Cell Carcinoma

Intervention / Treatment
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* Drug: BB-401


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Histologically or cytologically confirmed HNSCC Failed (or are ineligible/decline to receive) all available standard therapies Stable, treated brain metastases One target lesion defined as measurable (via RECIST v1.1), with a maximum longest diameter of 4 cm, and suitable for injection Eastern Cooperative Oncology Group (ECOG) 0-2 Agree to biopsies of a selected primary lesion and where possible of a second untreated lesion at baseline and end of treatment Ceased anti-cancer therapy for at least 3 weeks or 5 half-lives for chemotherapy based treatment regimen, or 4 weeks from any therapy with therapeutic biologicals or any type of investigational therapy Key Exclusion Criteria: Nasopharyngeal Carcinoma Concomitant anti-cancer therapy Unresolved toxicities from prior treatments Patients in whom the indicator lesion is at risk of hemorrhage or clinically significant swelling/inflammation

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BB-401<br>BB-401 Intratumoral injection | Drug: BB-401<br>* BB-401 1.92ug/mL Intratumoral Injections, every week for up to 8 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response (OR) of the injected tumor | Overall response is defined as Complete Response (CR) or Partial Response (PR) as evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee version 1.1 | Up to 20 months (estimated length of the study) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease Control Rate (DCR) | DCR is measured on a subset of subjects who achieve Stable Disease (SD), Partial Response (PR) or Complete Response (CR) at the End of Treatment visit. DCR will be defined as the proportion of these subjects that show absence of disease progression as evaluated using the RECIST v1.1 criteria | End of Treatment Visit (Week 9) through to study end (up to 18 months) | 
| Progression Free Survival (PFS) | PFS will be defined as the time from first treatment administration to first observation of documented disease progression as evaluated using the RECIST v1.1 criteria | Up to 20 months (estimated length of study) | 
| Overall Survival (OS) | OS will be defined as the time from first treatment administration through to death due to any cause | Up to 20 months (estimated length of study) | 
| Duration of Response (DoR) | DoR assessments will be performed on the subset of patients for who a CR, PR, or SD response is achieved. DoR will be measured from the time where the response criteria are first met, until the first date that progressive disease is objectively documented, or the date of death due to any cause | 20 months (estimated length of study) |