Nodular Basal Cell Carcinoma
Conditions
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 * Evaluation of the number of patients withdrawn from the trial * Investigator's global judgment of tolerability by means of a 6-point scale * Photographic documentation of the treatment area Exploratory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.
Interventions
DRUG0.06% Resiquimod Gel - A
single 60mg dose
DRUG0.06% Resiquimod Gel - B
single 100mg dose
DRUG0.06% Resiquimod Gel - C
shave biopsy of BCC followed by single 100mg dose
Sponsors
Spirig Pharma Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Histological Cure Rate | 8 weeks after a maximal treatment period of 4 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Clinical Clearance Rate | 8 weeks after the 4 weeks treatment period | — |
| Evaluation of Local Tolerability by Means of 5-point Scales | up to 12 weeks | local skin reactions as erythema, edema, erosion/ulceration, exudate, dryness, encrustation judged by investigator by means of 5-point scales (0 = absent, 1 = slight, 2 = moderate, 3 = severe, 4 = very severe). |
| Evaluation of Systemic Tolerability Based on Haematology and Blood Chemistry Values and Vital Signs | up to 12 weeks | — |
| Global Judgment of Tolerability by Investigator by Means of a 6-point Scale | 8 weeks after a maximal treatment period of 4 weeks | — |
Countries
Germany, Switzerland
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 0.06% Resiquimod Gel - A * 60 mg gel
* Once daily prior to normal sleeping hours
* 5x within 1 week (Monday to Friday) for 4 weeks (at maximum) or until clinical manifestation of skin erosion/crust formation
0.06% Resiquimod Gel - A | 1 |
| 0.06% Resiquimod Gel - B * 100 mg gel
* Once daily prior to normal sleeping hours
* 5x within 1 week (Monday to Friday) for 4 weeks (at maximum) or until clinical manifestation of skin erosion/crust formation
0.06% Resiquimod Gel - B | 3 |
| 0.06% Resiquimod Gel - C * 100 mg gel
* Once daily prior to normal sleeping hours
* 5x within 1 week (Monday to Friday) for 4 weeks (at maximum) or until clinical manifestation of skin erosion/crust formation
* The BCC will be pretreated. A shave biopsy (curettage or scraping off the tissue in a broad, superficial, tangential way) will be performed
0.06% Resiquimod Gel - C | 0 |
| Total | 4 |
Baseline characteristics
| Characteristic | 0.06% Resiquimod Gel - B | 0.06% Resiquimod Gel - A | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 participants | 0 participants | 0 participants |
| Age, Categorical >=65 years | 3 participants | 1 participants | 4 participants |
| Age, Categorical Between 18 and 65 years | 0 participants | 0 participants | 0 participants |
| Gender Female | 1 participants | 0 participants | 1 participants |
| Gender Male | 2 participants | 1 participants | 3 participants |
| Region of Enrollment Germany | 0 participants | 0 participants | 0 participants |
| Region of Enrollment Switzerland | 3 participants | 1 participants | 4 participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 1 / 1 | 1 / 3 | 0 / 0 |
| serious Total, serious adverse events | 0 / 1 | 1 / 3 | 0 / 0 |
Outcome results
Primary
Histological Cure Rate
Time frame: 8 weeks after a maximal treatment period of 4 weeks
Population: data were not collected for any of study participant.
Secondary
Complete Clinical Clearance Rate
Time frame: 8 weeks after the 4 weeks treatment period
Population: data were not collected for any of study participant.
Secondary
Evaluation of Local Tolerability by Means of 5-point Scales
local skin reactions as erythema, edema, erosion/ulceration, exudate, dryness, encrustation judged by investigator by means of 5-point scales (0 = absent, 1 = slight, 2 = moderate, 3 = severe, 4 = very severe).
Time frame: up to 12 weeks
Secondary
Evaluation of Systemic Tolerability Based on Haematology and Blood Chemistry Values and Vital Signs
Time frame: up to 12 weeks
Secondary
Global Judgment of Tolerability by Investigator by Means of a 6-point Scale
Time frame: 8 weeks after a maximal treatment period of 4 weeks