A Study of the Efficacy and Safety of ASN-002 in Adult Patients With Low-risk Nodular Basal Cell Carcinoma (ASN-002-001)

A Phase 1/2a Study of the Efficacy and Safety of ASN-002 Alone or in Combination With 5-FU in Adult Patients With Low-risk Nodular Basal Cell Carcinoma

The study evaluates whether ASN-002 is safe and effective in the treatment of nodular basal cell carcinoma (nBCC) in patients aged 18 years or over.

The participants will receive weekly injections of ASN-002 alone or in combination with 5-FU for 3 weeks and undergo surgical excision of the tumor.

Study Overview

• Status: Completed
• Conditions: Basal Cell Nevus Syndrome; Skin Neoplasm; Nodular Basal Cell Carcinoma of Skin
• Intervention / Treatment: Biological: ASN-002; Drug: 5-FU

Detailed Description

The primary purpose of this study is to determine whether ASN-002 alone or in combination with 5-FU is safe and effective in the treatment of nodular basal cell carcinoma (nBCC).

Patients aged 18 or over, who have been diagnosed with nodular Basal Cell Carcinoma (nBCC), may be eligible to join this study.

Study details:

ASN-002 is an immunotherapeutic product that is injected into the BCC spot to be treated. It is made from modified adenovirus serotype 5 (also called Ad5). Adenoviruses are common in nature worldwide and can cause mild colds and respiratory infections from which people usually recover without treatment. The Ad5 used in this study has been modified so that it cannot grow in the body or cause an infection. The modified Ad5 in this study will deliver artificially made genetic material into the cancerous and surrounding cells. This genetic material will produce human interferon which is normally produced by the body to stimulate the immune system. It is hoped that injected ASN-002 will cause the body's own cells to produce interferon and stimulate the immune system to attack the cancerous cells and reduce the size of or eliminate the nBCC. Participants will attend the study centre weekly for an injection of ASN-002 alone of in combination with 5-FU into the nBCC. The participants recruited will have 3 injections over 3 weeks, and then undergo surgical excision of the tumor.

Patient outcomes will then be assessed using a tumour sample collected during surgery and by the incidence of adverse events which occur throughout the study.

It is hoped that the findings of this trial will provide information on the safety and efficacy of using ASN-002 alone or in combination with 5-FU for nBCC, particularly for patients in whom the standard treatment of surgery is not possible or not recommended.

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Kogarah, New South Wales, Australia, 2217
      • St George Dermatology and Skin Cancer Centre
  • Queensland
    • Brisbane, Queensland, Australia, 4000
      • Siller Medical T/A Central Brisbane Dermatology
    • Brisbane, Queensland, Australia, 4102
      • Veracity Clinical Research
  • Victoria
    • Melbourne, Victoria, Australia, 3002
      • Sinclair Dermatology

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

Inclusion Criteria:

1. Low risk nodular basal cell carcinoma
2. Biopsy of any other skin tumor
3. Willingness to have injection therapy followed by surgery
4. Written informed consent

Exclusion Criteria:

1. No or only minimal symptoms
2. Known or suspected metastatic disease.
3. Pregnant or Lactating females
4. Clinically active or uncontrolled skin disease
5. Immunocompromised or receiving immunomodulating agent
6. treatment with psoralen plus Ultraviolet A or Ultraviolet B light therapy within 6 months
7. Any serious or active medical or psychiatric illness
8. Recreational or therapeutic drug or alcohol use
9. Taking any investigational product within 1 month of first dose of ASN- 002.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Cohort 1; Study Participants will receive ASN-002 at a dose 5X 10(10) vp, weekly injection in tumor nodules for 3 weeks.	Biological: ASN-002; ASN-002, is made of a suspension of recombinant adenoviral particles carrying a gene coding for the human interferon-gamma (IFN). Study participants will receive protocol defined dose of ASN-002 as intra-tumoral injections.
EXPERIMENTAL: Cohort 2; Study Participants will receive ASN-002 at a dose of 1.5X 10(11) vp weekly injection in tumor nodules for 3 weeks	Biological: ASN-002; ASN-002, is made of a suspension of recombinant adenoviral particles carrying a gene coding for the human interferon-gamma (IFN). Study participants will receive protocol defined dose of ASN-002 as intra-tumoral injections.
EXPERIMENTAL: Cohort 4; Study Participants will receive ASN- 002 at a dose of 3.0X 10(11) vp weekly injections in tumor nodules for 3 weeks.	Biological: ASN-002; ASN-002, is made of a suspension of recombinant adenoviral particles carrying a gene coding for the human interferon-gamma (IFN). Study participants will receive protocol defined dose of ASN-002 as intra-tumoral injections.
EXPERIMENTAL: Cohort 5; Study Participants will receive ASN- 002 at a dose of 2.25X 10(11) vp weekly injections in tumor nodules for 3 weeks.	Biological: ASN-002; ASN-002, is made of a suspension of recombinant adenoviral particles carrying a gene coding for the human interferon-gamma (IFN). Study participants will receive protocol defined dose of ASN-002 as intra-tumoral injections.
EXPERIMENTAL: Combination Cohorts; Study Participants will receive ASN- 002 at either dose of Cohorts II, IV or V in combination with a low dose 5-FU (1mg/2.5 mg/5mg/10mg or 25mg) weekly injections in tumor nodules for 3 weeks.	Biological: ASN-002; ASN-002, is made of a suspension of recombinant adenoviral particles carrying a gene coding for the human interferon-gamma (IFN). Study participants will receive protocol defined dose of ASN-002 as intra-tumoral injections.; Drug: 5-FU; 5-FU is chemotherapeutic agent used to treat various cancers.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidences of ASN-002 related Adverse Event in patients with previously untreated nBCC	changes in vital signs, adverse events, serious adverse events, laboratory abnormalities and withdrawals from study. Local skin and injection site reactions will be assessed in detail scoring erythema, ulceration, pain and overall severity as none, mild, moderate or severe.	Participants will be followed up for up to 6 months.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Microscopic clearance of the injected basal cell carcinoma.	Histological clearance (HC) will be defined as the absence of detectable evidence of BCC tumor cell nests in serial histological samples as determined by central pathology review.	Microscopic examinations of sample collected at 17weeks after the first dose.
Clinical Changes in size of nBCC tumor over time after treatment with ASN-002 alone or in combination with 5-FU	Change in nBCC lesion size will be assessed by investigator at baseline and then every 4 weeks until the surgical excision of BCC after ASN-002 therapy.	Change in nBCC will be assessed for up to 6 months from the first treatment visit.

Collaborators and Investigators

• Sponsor: Ascend Biopharmaceuticals Ltd
• Investigators: Principal Investigator: Lynda Spelman, Veracity Clinical Research Pty Ltd.; Principal Investigator: Rodney Sinclair, Sinclair Dermatology Pty Ltd; Principal Investigator: Gregory Siller, Siller Medical T/A Central Brisbane Dermatology

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 1, 2015
• Primary Completion (ACTUAL): November 1, 2017
• Study Completion (ACTUAL): November 1, 2017

Study Registration Dates

• First Submitted: September 10, 2015
• First Submitted That Met QC Criteria: September 14, 2015
• First Posted (ESTIMATE): September 15, 2015

Study Record Updates

• Last Update Posted (ACTUAL): June 6, 2018
• Last Update Submitted That Met QC Criteria: June 3, 2018
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Keywords: Low-Risk; Untreated nBCC
• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Cysts; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Stomatognathic Diseases; Bone Diseases; Neoplastic Syndromes, Hereditary; Jaw Diseases; Abnormalities, Multiple; Bone Diseases, Developmental; Odontogenic Cysts; Jaw Cysts; Bone Cysts; Neoplasms, Basal Cell; Carcinoma; Skin Neoplasms; Basal Cell Nevus Syndrome; Carcinoma, Basal Cell
• Other Study ID Numbers: ASN-002-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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