Clinical trial NCT01808950: Efficacy and Safety Trial of Topical Resiquimod Gel (0.06%) in Patients With Nodular Basal Cell Carcinoma
Official title: 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Nodular Basal Cell Carcinoma
Lead sponsor: Spirig Pharma Ltd.
Sponsor class: INDUSTRY
First posted: 2013-03-11
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: 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.

Detailed description: 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* Evaluation of the number of patients withdrawn from the trial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.

Eligibility criteria: 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.

Primary outcomes:
measure: Histological Cure Rate
timeFrame: 8 weeks after a maximal treatment period of 4 weeks
Secondary outcomes:
measure: Complete Clinical Clearance Rate
timeFrame: 8 weeks after the 4 weeks treatment period
measure: Evaluation of Local Tolerability by Means of 5-point Scales
timeFrame: up to 12 weeks
measure: Evaluation of Systemic Tolerability Based on Haematology and Blood Chemistry Values and Vital Signs
timeFrame: up to 12 weeks
measure: Global Judgment of Tolerability by Investigator by Means of a 6-point Scale
timeFrame: 8 weeks after a maximal treatment period of 4 weeks
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Hauttumorcentrum Charité (HTCC)
city: Zurich
country: Switzerland
facility: Universitaetsspital
Study officials:
affiliation: Clinical Dermatolgy Zurich
name: R Dummer, PrMD
role: PRINCIPAL_INVESTIGATOR
References: