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

Sponsor

Ascend Biopharmaceuticals Ltd (Industry)

Overall Status

Completed

CT.gov ID

NCT02550678

Collaborator

(none)

Enrollment

Locations

Arms

Actual Duration (Months)

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Basal Cell Nevus Syndrome Skin Neoplasm Nodular Basal Cell Carcinoma of Skin	Biological: ASN-002 Drug: 5-FU	Phase 1/Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

16 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Sep 1, 2015

Actual Primary Completion Date :

Nov 1, 2017

Actual Study Completion Date :

Nov 1, 2017

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

Incidences of ASN-002 related Adverse Event in patients with previously untreated nBCC [Participants will be followed up for up to 6 months.]
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.

Secondary Outcome Measures

Microscopic clearance of the injected basal cell carcinoma. [Microscopic examinations of sample collected at 17weeks after the first dose.]
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.
Clinical Changes in size of nBCC tumor over time after treatment with ASN-002 alone or in combination with 5-FU [Change in nBCC will be assessed for up to 6 months from the first treatment visit.]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	St George Dermatology and Skin Cancer Centre	Kogarah	New South Wales	Australia	2217
2	Siller Medical T/A Central Brisbane Dermatology	Brisbane	Queensland	Australia	4000
3	Veracity Clinical Research	Brisbane	Queensland	Australia	4102
4	Sinclair Dermatology	Melbourne	Victoria	Australia	3002

Sponsors and Collaborators

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