A phase I study of the Dz13 drug targeting the c-Jun gene in subjects with skin cance (nodular Basal Cell Carcinoma).

A phase I study to determine the safety and tolerability of Dz13 DNAzyme targeting c-Jun in subjects with nodular Basal Cell Carcinoma.

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12610000162011

Acronym

DISCOVER

Enrollment

Registered

2010-02-19

Start date

2010-09-06

Completion date

Unknown

Last updated

2020-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

The purpose of this study is to test the safety of a new treatment (Dz13 combined with DOPE and DOTAP) for nodular BCC, a form of skin cancer. Dz13 is a small piece of DNA which binds to an mRNA molecule in the cell which in turn produces a specific protein known as c-Jun. The c-Jun gene is known to be abnormally active in many types of cancer cells, including BCC, causing the abnormal production of the c-Jun protein molecule. The c-Jun protein is involved in 'switiching on' other genes involved in cell growth, resulting in growth and spread of tumour cells. Dz13 binds to the c-Jun mRNA and chops it into two pieces, which disrupts production of the c-Jun protein and limits the growth and spread of the tumour. c-Jun is present at very low levels in normal adult tissues and is mostly expressed at high levels in tumour tissue. It is hypothesised that Dz13 will be able to stop the growth and spread of BCC and other skin tumours without significant toxicity. Dz13 has been shown in animal studies to be well tolerated at doses 35-fold higher than the highest dose that will be used in this study.

Interventions

Dz13 DNAzyme complexed with the lipids DOPE and DOTAP administered as a single intratumoural injection. Three dose cohorts of 10 mcg, 30 mcg and 100 mcg Dz13.

Study design

Allocation

Non-randomised trial

Intervention model

Other

Primary purpose

Treatment

Masking

Blinded (masking used)

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

Histologically proven nodular BCC. Measurable disease of 8-16 mm located on trunk or limbs. Presence of dividing cells as identified histologically.

Exclusion criteria

Women of childbearing potential. Prior or co-existing malignancy. Radiotherapy to >30% bone marrow in previous three months. Known genetic predisposition to skin cancer. Clinically significant non-malignant disease. Current immunosuppression. History of immune-mediated thrombocytopenia or other platelet disease. History of drug abuse. Enrollment in another clinical study using another investigational agent.

Outcome results