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

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

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.

Study Overview

• Status: Completed
• Conditions: Head and Neck Squamous Cell Carcinoma
• Intervention / Treatment: Drug: BB-401

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia
      • The Chris O'Brien Lifehouse
  • South Australia
    • Elizabeth Vale, South Australia, Australia
      • Calvary Central Districts Hospital
• Russian Federation
  • Irkutsk, Russian Federation
    • Irkutsk Oncology Center
  • Krasnodar, Russian Federation
    • Clinical Oncology Dispensary #1
  • Leningrad Region, Russian Federation
    • Leningrad Regional Oncology Dispensary
  • Saint Petersburg, Russian Federation
    • Saint Petersburg City Oncology Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Benitec BioPharma Ltd

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2018
• Primary Completion (Actual): December 12, 2018
• Study Completion (Actual): April 6, 2020

Study Registration Dates

• First Submitted: February 5, 2018
• First Submitted That Met QC Criteria: February 8, 2018
• First Posted (Actual): February 14, 2018

Study Record Updates

• Last Update Posted (Actual): April 15, 2020
• Last Update Submitted That Met QC Criteria: April 13, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Head and Neck Neoplasms; Neoplasms, Squamous Cell; Carcinoma; Carcinoma, Squamous Cell; Squamous Cell Carcinoma of Head and Neck
• Other Study ID Numbers: BB-401-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: De-identified individual participant data for primary and secondary outcome measures will be made available for the Interim Analysis and routinely through-out the study for the Data Safety Monitoring Board
• IPD Sharing Time Frame: Data will be available within 6-8 weeks from data cut off/lock
• IPD Sharing Access Criteria: Data will only be available to the Data Safety Monitoring Board and Interim Analysis members
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR); Analytic Code

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No