Trial Outcomes & Findings for Pilot LDE225 in Locally Advanced or Metastatic BCC + Previously Tx Non-LDE225 Smoothened Inhibitors (NCT NCT01529450)
NCT ID: NCT01529450
Last Updated: 2017-01-16
Results Overview
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
11 participants
Primary outcome timeframe
End of treatment or at time of disease progression (up to 58 weeks)
Results posted on
2017-01-16
Participant Flow
Participant milestones
| Measure |
Refractory Group
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
8
|
|
Overall Study
COMPLETED
|
3
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pilot LDE225 in Locally Advanced or Metastatic BCC + Previously Tx Non-LDE225 Smoothened Inhibitors
Baseline characteristics by cohort
| Measure |
Refractory Group
n=3 Participants
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
n=6 Participants
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
65 years
STANDARD_DEVIATION 24 • n=5 Participants
|
54 years
STANDARD_DEVIATION 9 • n=7 Participants
|
57 years
STANDARD_DEVIATION 15 • n=5 Participants
|
|
Gender
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Gender
Male
|
2 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
6 participants
n=7 Participants
|
9 participants
n=5 Participants
|
|
Size of disease by RECIST criteria
|
3.8 centimeters
STANDARD_DEVIATION 1.3 • n=5 Participants
|
4.2 centimeters
STANDARD_DEVIATION 2.4 • n=7 Participants
|
4.0 centimeters
STANDARD_DEVIATION 2.0 • n=5 Participants
|
PRIMARY outcome
Timeframe: End of treatment or at time of disease progression (up to 58 weeks)Population: Progression is defined using RECIST version 1.0 as a 20% increase in the sum of the longest diameter of target lesions, or a measureable increase in a non-target lesion or the appearance of new lesion.
Outcome measures
| Measure |
Refractory Group
n=3 Participants
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
n=6 Participants
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
|---|---|---|
|
Progression Free Survival (PFS) of All Participants
|
6 weeks
Interval 3.0 to 12.0
|
36 weeks
Interval 14.0 to 58.0
|
SECONDARY outcome
Timeframe: Assessed on day 1Population: Participants with tissue available for screening were analyzed.
Following consent, historical biopsy samples were analyzed for molecular markers associated with clinical response. Tumors were analyzed and present of Smooth mutation (SMO \[genetic changes which influence Ki 67 and Gli levels\]). was determined. The number of participants with and without SMO were reported by clinical outcome (SD = stable disease; PD = progressive disease).
Outcome measures
| Measure |
Refractory Group
n=7 Participants
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
|---|---|---|
|
Molecular Markers Associated With Clinical Response
SMO Present (outcome SD)
|
1 participants
|
—
|
|
Molecular Markers Associated With Clinical Response
SMO absent (outcome SD)
|
1 participants
|
—
|
|
Molecular Markers Associated With Clinical Response
SMO Present (outcome PD)
|
4 participants
|
—
|
|
Molecular Markers Associated With Clinical Response
SMO absent (outcome PD)
|
1 participants
|
—
|
Adverse Events
Refractory Group
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Resistance Developed Group
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Refractory Group
n=3 participants at risk
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
n=6 participants at risk
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
|---|---|---|
|
Nervous system disorders
Altered mental status
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6 • Number of events 1
Participants with evaluable data are included for Adverse Events
|
Other adverse events
| Measure |
Refractory Group
n=3 participants at risk
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
Resistance Developed Group
n=6 participants at risk
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
LDE225: 800-mg (4 200-mg capsules/day) capsule
|
|---|---|---|
|
Cardiac disorders
palpitations
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Ear and labyrinth disorders
ear canal stenosis
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Ear and labyrinth disorders
ear drainage
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Ear and labyrinth disorders
hearing loss
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
appetite decrease
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
dehydration
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
diarrhea
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
nausea
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
33.3%
2/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
taste disturbance
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Gastrointestinal disorders
vomit
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
General disorders
weight loss
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Infections and infestations
candidiasis
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Injury, poisoning and procedural complications
ecchymosis
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Injury, poisoning and procedural complications
laceration
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Investigations
elevated creatine kinase
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Musculoskeletal and connective tissue disorders
hip fracture
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Musculoskeletal and connective tissue disorders
muscle cramps
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
66.7%
4/6
Participants with evaluable data are included for Adverse Events
|
|
Musculoskeletal and connective tissue disorders
restless arms and legs
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Musculoskeletal and connective tissue disorders
rhabdomyolysis
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
neoplasm
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Nervous system disorders
altered mental status
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Nervous system disorders
migraine
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Nervous system disorders
somnolence
|
33.3%
1/3
Participants with evaluable data are included for Adverse Events
|
0.00%
0/6
Participants with evaluable data are included for Adverse Events
|
|
Psychiatric disorders
agitation
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/3
Participants with evaluable data are included for Adverse Events
|
16.7%
1/6
Participants with evaluable data are included for Adverse Events
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place