Efficacy and Safety Trial of Topical Resiquimod Gel (0.06%) in Patients With Nodular Basal Cell Carcinoma

Bi-center, Open Label, Non-comparative Trial Exploring Efficacy and Safety of Topical Resiquimod Gel (0.06%) in Patients With Nodular Basal Cell Carcinoma (nBCC)

The primary objective is the observation and description of the preliminary efficacy of resiquimod gel 0.06% on a single nodular basal cell carcinoma (nBCC) in a small group of patients.

efficacy assessments:

• Histopathological findings based on the biopsies of the primary tumor location and the tissue excision at the end of trial (histological cure).
• Description of the clinical-therapeutic effect of resiquimod on nBCC (nodular-basal cell carcinoma) by visual inspection (clinical evaluation of treatment area and assessment of complete clinical clearance)
• RNA-analysis (analysis of gene expressions for cytokines, cytotoxic and apoptotic signals)
• Investigator's global judgment of efficacy by means of a 7-point scale

Safety assessments:

• Evaluation of Adverse Events (AEs) and Serious Adverse Events (SAEs)
• Evaluation of local tolerability (local skin reactions as erythema, edema, erosion/ulceration, exsudate, dryness, encrustation) by means of symptom scoring scales (0 = absent, 1 = slight, 2 = moderate, 3 = severe, 4 = very severe).
• Evaluation of systemic tolerability [hematology (erythrocytes, leucocytes including neutrophils, hemoglobin, hematocrit, thrombocytes), blood chemistry (alkaline phosphatase, bilirubin, aspartate transaminase (ASAT), alanine transaminase (ALAT), serum creatinine), vital signs]. The thresholds concerning laboratory abnormalities that determine patient's discontinuation from trial were predefined upfront.
• Evaluation of the number of patients withdrawn from the trial
• Investigator's global judgment of tolerability by means of a 6-point scale
• Photographic documentation of the treatment area

Exploratory parameter:

• C-reactive protein (CRP)
• Interferon-alpha, interleukin-6, interleukin-12, interferon-gamma, TNF-alpha (up-regulation of gene expression)
• Immunohistochemistry and characterization of cell types (CD8, T-cells, macrophages, dendritic cells)
• In addition, blood serum samples will be preserved and frozen for later tests that will be specified to the patients. The preserved material will be stored for a maximum of 2 years.

Inclusion Criteria:

• Signed consent form.
• Male or non-pregnant, non-lactating female, ≥ 18 years.
• Must have a previously untreated, histologically confirmed nBCC on head, neck, trunk or arms.
• nBCC must not be larger than 20 mm in diameter and must be less than 5 mm in depth.
• Willing and able to participate in the trial as an outpatient and comply with all trial requirements.

Exclusion Criteria:

• nBCC located close to or at mouth or eyes.
• Patients who have had an organ transplant.
• Known autoimmune disorder (especially psoriasis), impaired immune system (e.g. HIV), known thyroid abnormalities, known depression.
• An open wound or an infection in treatment area.
• Dermatological disease or condition (e.g. rosacea, atopic dermatitis, eczema) in the treatment or surrounding area that might impair trial assessments.
• Evidence of an active infection or systemic cancer.
• Flu or flu-like symptoms (including general indisposition, fever, nausea, muscle pain, chills) within a week before start of the trial.
• Known allergy or hypersensitivity to any of the trial gel ingredients.
• Evidence of unstable or uncontrolled clinically significant medical conditions as determined by the investigator (e.g., renal or hepatic disease).
• Current alcohol abuse or chemical dependency as assessed by the investigator.
• Patient who is detained or committed to an institution by a law court or by legal authorities.
• Participation in another clinical trial within one month before start of the trial.