Sporadic Superficial and Nodular Skin Basal Cell Carcinomas
Conditions
Keywords
Basal cell carcinomas,, sporadic,, superficial and nodular,, skin
Brief summary
This was a double-blinded, randomized, vehicle-controlled study in sporadic superficial BCC (sBCC) and nodular BCC (nBCC) patients which consisted of a 21-day screening period, a treatment period of 6 weeks (topical 0.75% LDE225 cream application b.i.d) ending with post treatment biopsies, as safety visit one week after final study drug administration (Day 50), a visit on Day 83 for excision of the treated BCC, and an end of study evaluation (Day 90).
Interventions
DRUGLDE225 0.75%
0.75% cream
DRUGVehicle
matching placebo cream to 0.75% LDE225 cream
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Patients with one histologically confirmed superficial and nodular basal cell carcinoma (8-20 mm) eligible for surgical excision on selected body areas (scalp, arm, frontal trunk, posterior trunk, upper legs)
Exclusion criteria
* Previous treatment of the sBCC that are selected for treatment. * Any systemic treatment which is known to affect BCCs esp. cytostatic treatments, retinoids and photodynamic treatments. * Dark-skinned persons whose skin color prevents readily assessment of skin reactions Other protocol defined Incl./Excl. criteria may apply.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Evaluation of sBCCs Tumors | Day 43 | The clinical response parameters were defined as (i) complete response (i.e., there is no longer any visible evidence of a lesion consistent with BCC at this site), (ii) partial response (i.e., although a BCC still remains at this site, it has demonstrated a visible decrease in size compared with baseline), and (iii) no response / worsening (i.e., the BCC has not demonstrated any visible decrease in size compared with baseline). |
Countries
Australia, Austria
Participant flow
Recruitment details
Twenty-five participants were enrolled into the trial. However, one participant was excluded from the efficacy analysis. Therefore, the enrollment number differs from the number started below.
Participants by arm
| Arm | Count |
|---|---|
| LDE225 0.75% Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks. | 16 |
| Vehicle Participants topically applied matching placebo cream twice daily for 6 weeks. | 8 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | LDE225 0.75% | Vehicle | Total |
|---|---|---|---|
| Age, Continuous | 63 Years STANDARD_DEVIATION 7.3 | 63 Years STANDARD_DEVIATION 7.5 | 63 Years STANDARD_DEVIATION 7.4 |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 8 Participants |
| Sex: Female, Male Male | 12 Participants | 4 Participants | 16 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 | 8 / 16 | 5 / 8 | 1 / 1 |
| serious Total, serious adverse events | 0 / 16 | 0 / 8 | 0 / 1 |
Outcome results
Primary
Clinical Evaluation of sBCCs Tumors
The clinical response parameters were defined as (i) complete response (i.e., there is no longer any visible evidence of a lesion consistent with BCC at this site), (ii) partial response (i.e., although a BCC still remains at this site, it has demonstrated a visible decrease in size compared with baseline), and (iii) no response / worsening (i.e., the BCC has not demonstrated any visible decrease in size compared with baseline).
Time frame: Day 43
Population: Efficacy analysis set: the efficacy analysis set included all randomized participants with evaluable data.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| LDE225 0.75% | Clinical Evaluation of sBCCs Tumors | Complete response | 3 Participants |
| LDE225 0.75% | Clinical Evaluation of sBCCs Tumors | Partial response | 9 Participants |
| LDE225 0.75% | Clinical Evaluation of sBCCs Tumors | No response | 4 Participants |
| Vehicle | Clinical Evaluation of sBCCs Tumors | Complete response | 1 Participants |
| Vehicle | Clinical Evaluation of sBCCs Tumors | Partial response | 4 Participants |
| Vehicle | Clinical Evaluation of sBCCs Tumors | No response | 3 Participants |