Trial Outcomes & Findings for To Evaluate the Safety, Local Tolerability, PK and PD of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas (sBCC) (NCT NCT01033019)
NCT ID: NCT01033019
Last Updated: 2015-10-30
Results Overview
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).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
Day 43
Results posted on
2015-10-30
Participant Flow
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.
Participant milestones
| Measure |
LDE225 0.75%
Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
|
Vehicle
Participants topically applied matching placebo cream twice daily for 6 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
8
|
|
Overall Study
COMPLETED
|
15
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
LDE225 0.75%
Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
|
Vehicle
Participants topically applied matching placebo cream twice daily for 6 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
To Evaluate the Safety, Local Tolerability, PK and PD of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas (sBCC)
Baseline characteristics by cohort
| Measure |
LDE225 0.75%
n=16 Participants
Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
|
Vehicle
n=8 Participants
Participants topically applied matching placebo cream twice daily for 6 weeks.
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
63 Years
STANDARD_DEVIATION 7.3 • n=99 Participants
|
63 Years
STANDARD_DEVIATION 7.5 • n=107 Participants
|
63 Years
STANDARD_DEVIATION 7.4 • n=206 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Day 43Population: Efficacy analysis set: the efficacy analysis set included all randomized participants with evaluable data.
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).
Outcome measures
| Measure |
LDE225 0.75%
n=16 Participants
Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
|
Vehicle
n=8 Participants
Participants topically applied matching placebo cream twice daily for 6 weeks.
|
|---|---|---|
|
Clinical Evaluation of sBCCs Tumors
No response
|
4 Participants
|
3 Participants
|
|
Clinical Evaluation of sBCCs Tumors
Complete response
|
3 Participants
|
1 Participants
|
|
Clinical Evaluation of sBCCs Tumors
Partial response
|
9 Participants
|
4 Participants
|
Adverse Events
LDE225 0.75% - sBCC
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Vehicle - sBCC
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
LDE225 0.75% - nBCC
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
LDE225 0.75% - sBCC
n=16 participants at risk
Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks.
|
Vehicle - sBCC
n=8 participants at risk
Participants topically applied matching placebo cream twice daily for 6 weeks.
|
LDE225 0.75% - nBCC
n=1 participants at risk
|
|---|---|---|---|
|
Investigations
Glomerular filtration rate increased
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
100.0%
1/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Gastrointestinal disorders
Gingival cyst
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Gastrointestinal disorders
Gingivitis
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
General disorders
Application site dermatitis
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
General disorders
Application site pruritus
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
General disorders
Local swelling
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Infections and infestations
Acute tonsillitis
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Infections and infestations
Application site folliculitis
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Infections and infestations
Gastrointestinal infection
|
12.5%
2/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Infections and infestations
Oral herpes
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Injury, poisoning and procedural complications
Incision site pain
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Injury, poisoning and procedural complications
Post procedural haematoma
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Investigations
Blood creatinine increased
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Investigations
Blood glucose increased
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Investigations
Blood pressure increased
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Investigations
Body temperature increased
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
100.0%
1/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Investigations
Glucose urine present
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Musculoskeletal and connective tissue disorders
Polyarthritis
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Nervous system disorders
Headache
|
0.00%
0/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
12.5%
1/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
100.0%
1/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Renal and urinary disorders
Dysuria
|
6.2%
1/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
12.5%
2/16
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/8
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
0.00%
0/1
The nBCC participant from the LDE225 75% group was analyzed for safety as a separate arm.
|
Additional Information
Study Director
Novartis
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
- Publication restrictions are in place
Restriction type: OTHER