Superficial Basal Cell Carcinoma
Conditions
Keywords
Double-Blind, Placebo, Topical, Treatment, Dose-Ranging, sBCC
Brief summary
The purpose of this study is to determine the safety and tolerability of pharmaceutical compound (API 31510) topical cream topically applied to superficial basal cell carcinomas to obtain preliminary efficacy data for the treatment of superficial basal cell carcinomas by API 31510 topical cream. The study population will include one hundred and sixty (160) otherwise healthy male or female adults with one or more histologically confirmed superficial basal cell carcinoma lesions. One target lesion with an area of less than one inch will be designated for study treatment.
Interventions
DRUGAPI 31510 3% Topical Cream
Topical treatment 3% active cream applied to the lesion
DRUGAPI 31510 1.5% Topical Cream
Topical treatment 1.5% active cream applied to the lesion
OTHERPlacebo
Topical treatment placebo applied to the lesion
Sponsors
BPGbio
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female adults ≥ 18 years of age * Primary, histologically confirmed sBCC with a minimum area of 0.5 cm2 and with a maximum diameter of 2.0 cm target sBCC lesion suitable for excision * Histological diagnosis made no more than 4 weeks prior to the screening visit * Histological biopsy removed 25% or less of the target lesion * No other dermatological disease in the sBCC target site or surrounding area * Willing to refrain from using non-approved lotions or creams on the target site and surrounding area during the treatment period. Willing to refrain from washing the treated area for at least 8 hours following the application of study medication * Willing to refrain from exposure to direct sunlight or ultraviolet light and to avoid the use of tanning parlors for the duration of the study * Laboratory values for the tests listed in the Study Schedule on page 2 within the reference ranges as defined by the central laboratory, or out of range test results that are clinically acceptable to the investigator. * Ability to follow study instructions and likely to complete all study requirements * Written informed consent obtained, including consent for tissue to be examined and stored by the central dermatopathologist * Written consent to allow photographs of the target sBCC lesion to be used as part of the study data * For females of childbearing potential, a negative pregnancy test at screening and using an acceptable form of birth control (oral/implant/injectable/transdermal contraceptives, intrauterine device, condom, diaphragm, abstinence, or a monogamous relationship with a partner who has had a vasectomy)
Exclusion criteria
* Pregnant or lactating * Presence of known or suspected systemic cancer * Histological evidence of nBCC, SCC, or any other tumor in the biopsy specimen * Histological evidence of severe squamous metaplasia, infiltrative, desomoplastic or micronodular growth patterns in the biopsy specimen * History of recurrence of the target sBCC lesion * Evidence of dermatological disease or confounding skin condition in the treatment area, e.g., SCC, actinic keratosis, rosacea, psoriasis, atopic dermatitis, eczema, xeroderma pigmentosa * Concurrent disease or treatment that suppresses the immune system * Chronic medical condition that in the judgment of the investigator(s) would interfere with the performance of the study or would place the patient at undue risk * Known sensitivity to any of the ingredients in the study medication * Use of a tanning parlor or other excessive or prolonged exposure to ultraviolet light or direct sunlight during the course of the study * Treatment with systemic chemotherapeutic agents within the 6 months prior to the screening visit * Use of systemic retinoids within the 6 months prior to the screening period * Treatment with systemic immunomodulators or immunosuppressants within the 6 months prior to the screening period * Use of topical immunomodulators within 2 cm of the target BCC lesion within the 4 weeks prior to the screening period * Treatment with the following topical agents within the 4 weeks prior to the screening visit: levulanic acid, 5-fluorouracil, coricosteroids, retinoids, diclofenac, hyaluronic acid, imiquimod * Undergone a facial resurfacing procedure, i.e., chemical peel, laser resurfacing, dermabrasion, within the 6 months prior to the screening visit, if the target sBCC lesion is on the face * Treatment with liquid nitrogen, surgical excision or curettage within 2 cm of the target sBCC lesion during the 4 weeks prior to the screening visit * Elective surgery within 4 weeks prior to the screening visit, during the study, or 4 weeks after the treatment period * Evidence of current chronic alcohol or drug abuse * Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of the screening visit * In the investigator's opinion, evidence of unwillingness, or inability to follow the restrictions of the protocol and complete the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Complete Response | 10 weeks | To determine the number of participants with a complete response, defined as a negative histological examination of the sBCC lesion at Week 10 (4 weeks post-treatment). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Partial Response | 10 weeks | To determine the number of participants with a partial response, defined as a clinically significant decrease (ie, at least 50%) in the area of the sBCC lesion, computed as the product of the two principal diameters at Week 10 (4 weeks post-treatment) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 3% BID 3.0% active twice daily (BID) | 47 |
| 3% QD 3.0% active cream once daily (QD) plus placebo cream QD | 45 |
| 1.5% QD 1.5% active cream once daily (QD) plus placebo cream QD | 48 |
| Placebo Placebo cream twice daily (BID) | 46 |
| Total | 186 |
Baseline characteristics
| Characteristic | 3% BID | 3% QD | 1.5% QD | Placebo | Total |
|---|---|---|---|---|---|
| Age, Continuous | 62.6 years | 61.2 years | 60.4 years | 62.8 years | 61.7 years |
| Sex: Female, Male Female | 17 Participants | 20 Participants | 23 Participants | 16 Participants | 76 Participants |
| Sex: Female, Male Male | 30 Participants | 25 Participants | 25 Participants | 30 Participants | 110 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 14 / 47 | 22 / 45 | 18 / 48 | 26 / 46 |
| serious Total, serious adverse events | 3 / 47 | 0 / 45 | 0 / 48 | 0 / 46 |
Outcome results
Primary
Number of Participants With Complete Response
To determine the number of participants with a complete response, defined as a negative histological examination of the sBCC lesion at Week 10 (4 weeks post-treatment).
Time frame: 10 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| 3% Active Cream BID | Number of Participants With Complete Response | 9 Participants |
| 3% Active Cream QD | Number of Participants With Complete Response | 5 Participants |
| 1.5% QD | Number of Participants With Complete Response | 7 Participants |
| Placebo BID | Number of Participants With Complete Response | 9 Participants |
Secondary
Number of Participants With Partial Response
To determine the number of participants with a partial response, defined as a clinically significant decrease (ie, at least 50%) in the area of the sBCC lesion, computed as the product of the two principal diameters at Week 10 (4 weeks post-treatment)
Time frame: 10 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| 3% Active Cream BID | Number of Participants With Partial Response | 11 Participants |
| 3% Active Cream QD | Number of Participants With Partial Response | 8 Participants |
| 1.5% QD | Number of Participants With Partial Response | 13 Participants |
| Placebo BID | Number of Participants With Partial Response | 13 Participants |