Trial Outcomes & Findings for A Pre-surgical Study of LDE225 in Men With High-risk Localized Prostate Cancer (NCT NCT02111187)
NCT ID: NCT02111187
Last Updated: 2019-03-07
Results Overview
This was defined as the number of patients who achieved at least a two-fold reduction in GLI1 expression in post-treatment vs. pre-treatment tumor tissues.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
14 participants
Primary outcome timeframe
Up to 3 Years
Results posted on
2019-03-07
Participant Flow
Participant milestones
| Measure |
LDE225 (Arm1)
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
7
|
|
Overall Study
COMPLETED
|
7
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Pre-surgical Study of LDE225 in Men With High-risk Localized Prostate Cancer
Baseline characteristics by cohort
| Measure |
LDE225 (Arm1)
n=7 Participants
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 Participants
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
Total
n=14 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
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
caucasian
|
7 participants
n=5 Participants
|
6 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African-American
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
14 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3 YearsThis was defined as the number of patients who achieved at least a two-fold reduction in GLI1 expression in post-treatment vs. pre-treatment tumor tissues.
Outcome measures
| Measure |
LDE225 (Arm1)
n=7 Participants
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 Participants
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Change From Baseline in Tissue Gli1 Expression Levels Using qRT-PCR Analysis in Each Group (LDE225 and Observation)
|
6 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 3 yearsTo determine whether presurgical treatment with LDE225 can exert a pathological effect on high-risk tumors (i.e. increase apoptosis, decrease proliferation).
Outcome measures
| Measure |
LDE225 (Arm1)
n=7 Participants
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 Participants
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Percentage of Participants With a Pathological Effect of Presurgical Treatment With LDE225
Pathologic Gleason Sum 9
|
5 Participants
|
6 Participants
|
|
Percentage of Participants With a Pathological Effect of Presurgical Treatment With LDE225
Pathologic Gleason Sum 10
|
1 Participants
|
0 Participants
|
|
Percentage of Participants With a Pathological Effect of Presurgical Treatment With LDE225
Pathologic Gleason Sum 7
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 3 yearsTo evaluate whether presurgical treatment with LDE225 diminishes the risk of PSA recurrence following prostatectomy.
Outcome measures
| Measure |
LDE225 (Arm1)
n=7 Participants
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 Participants
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Effect of LDE225 on PSA Recurrence Following Prostatectomy
|
12.0 ng/ml
Interval 3.2 to 29.7
|
7.4 ng/ml
Interval 2.4 to 10.4
|
SECONDARY outcome
Timeframe: Up to 3 yearsSafety and tolerability, including any drug-related toxicities of Sonidegib, were reported via CTCAE version 4.0.
Outcome measures
| Measure |
LDE225 (Arm1)
n=7 Participants
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 Participants
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Number of Participants With Adverse Events in Each Group (LDE225 and Observation)
|
3 Participants
|
0 Participants
|
Adverse Events
LDE225 (Arm1)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Observation Arm (Arm2)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
LDE225 (Arm1)
n=7 participants at risk
Treatment arm (Arm 1) will receive LDE225 by mouth 800 mg daily for 4 weeks (+/- 3 days)
LDE225
|
Observation Arm (Arm2)
n=7 participants at risk
Observation Arm (Arm2) will receive no treatment prior to prostatectomy.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
42.9%
3/7 • Number of events 3 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Musculoskeletal and connective tissue disorders
Increased CK level
|
28.6%
2/7 • Number of events 3 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Hepatobiliary disorders
AST/ALT elevated
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
General disorders
Fatigue
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Endocrine disorders
Dry mouth
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Endocrine disorders
dysgeusia
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
|
Musculoskeletal and connective tissue disorders
musculoskeletal pain
|
14.3%
1/7 • Number of events 1 • 1 year and 6 months
|
0.00%
0/7 • 1 year and 6 months
|
Additional Information
Emmanuel Antonarakis
Associate professor of oncology and urology
Phone: 443-287-0553
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place