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Trial record 57 of 109 for:    hedgehog

Pilot LDE225 in Locally Advanced or Metastatic BCC + Previously Tx Non-LDE225 Smoothened Inhibitors

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ClinicalTrials.gov Identifier: NCT01529450
Recruitment Status : Completed
First Posted : February 8, 2012
Results First Posted : November 2, 2016
Last Update Posted : January 16, 2017
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
Anne Chang, Stanford University

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Basal Cell Carcinoma
Intervention Drug: LDE225
Enrollment 11

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Refractory Group Resistance Developed Group
Hide Arm/Group Description

Patients previously treated with non-LDE225 Smo inhibitor who were refractory.

LDE225: 800-mg (4 200-mg capsules/day) capsule

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

Period Title: Overall Study
Started 3 8
Completed 3 6
Not Completed 0 2
Arm/Group Title Refractory Group Resistance Developed Group Total
Hide Arm/Group Description

Patients previously treated with non-LDE225 Smo inhibitor who were refractory.

LDE225: 800-mg (4 200-mg capsules/day) capsule

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 of all reporting groups
Overall Number of Baseline Participants 3 6 9
Hide Baseline Analysis Population Description
Participants with evaluable data are included for Baseline Characteristics
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 6 participants 9 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
3
 100.0%
4
  66.7%
7
  77.8%
>=65 years
0
   0.0%
2
  33.3%
2
  22.2%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 6 participants 9 participants
65  (24) 54  (9) 57  (15)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 6 participants 9 participants
Female
1
  33.3%
0
   0.0%
1
  11.1%
Male
2
  66.7%
6
 100.0%
8
  88.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants 6 participants 9 participants
3 6 9
Size of disease by RECIST criteria  
Mean (Standard Deviation)
Unit of measure:  Centimeters
Number Analyzed 3 participants 6 participants 9 participants
3.8  (1.3) 4.2  (2.4) 4.0  (2.0)
1.Primary Outcome
Title Progression Free Survival (PFS) of All Participants
Hide Description [Not Specified]
Time Frame End of treatment or at time of disease progression (up to 58 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Refractory Group Resistance Developed Group
Hide Arm/Group Description:

Patients previously treated with non-LDE225 Smo inhibitor who were refractory.

LDE225: 800-mg (4 200-mg capsules/day) capsule

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 Number of Participants Analyzed 3 6
Median (95% Confidence Interval)
Unit of Measure: weeks
6
(3 to 12)
36
(14 to 58)
2.Secondary Outcome
Title Molecular Markers Associated With Clinical Response
Hide Description 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).
Time Frame Assessed on day 1
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants with tissue available for screening were analyzed.
Arm/Group Title All Participants
Hide Arm/Group Description:
Participants received LDE225: 800-mg (4 200-mg capsules/day) capsule
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
SMO Present (outcome SD) 1
SMO absent (outcome SD) 1
SMO Present (outcome PD) 4
SMO absent (outcome PD) 1
Time Frame [Not Specified]
Adverse Event Reporting Description Participants with evaluable data are included for Adverse Events
 
Arm/Group Title Refractory Group Resistance Developed Group
Hide Arm/Group Description

Patients previously treated with non-LDE225 Smo inhibitor who were refractory.

LDE225: 800-mg (4 200-mg capsules/day) capsule

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

All-Cause Mortality
Refractory Group Resistance Developed Group
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Refractory Group Resistance Developed Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/3 (0.00%)      1/6 (16.67%)    
Nervous system disorders     
Altered mental status * 1  0/3 (0.00%)  0 1/6 (16.67%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.3)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Refractory Group Resistance Developed Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      6/6 (100.00%)    
Cardiac disorders     
palpitations  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
Ear and labyrinth disorders     
ear canal stenosis  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
ear drainage  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
hearing loss  1 [2]  1/3 (33.33%)  0/6 (0.00%) 
Gastrointestinal disorders     
appetite decrease  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
dehydration  1 [3]  0/3 (0.00%)  1/6 (16.67%) 
diarrhea  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
nausea  1 [3]  0/3 (0.00%)  2/6 (33.33%) 
taste disturbance  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
vomit  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
General disorders     
weight loss  1 [2]  1/3 (33.33%)  0/6 (0.00%) 
Infections and infestations     
candidiasis  1 [2]  1/3 (33.33%)  0/6 (0.00%) 
Injury, poisoning and procedural complications     
ecchymosis  1 [2]  0/3 (0.00%)  1/6 (16.67%) 
laceration  1 [2]  0/3 (0.00%)  1/6 (16.67%) 
Investigations     
elevated creatine kinase  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
Musculoskeletal and connective tissue disorders     
hip fracture  1 [3]  0/3 (0.00%)  1/6 (16.67%) 
muscle cramps  1 [1]  0/3 (0.00%)  4/6 (66.67%) 
restless arms and legs  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
rhabdomyolysis  1 [3]  0/3 (0.00%)  1/6 (16.67%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
neoplasm  1 [2]  1/3 (33.33%)  0/6 (0.00%) 
Nervous system disorders     
altered mental status  1 [3]  0/3 (0.00%)  1/6 (16.67%) 
migraine  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
somnolence  1 [1]  1/3 (33.33%)  0/6 (0.00%) 
Psychiatric disorders     
agitation  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
Respiratory, thoracic and mediastinal disorders     
cough  1 [1]  0/3 (0.00%)  1/6 (16.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.3)
[1]
Grade 1
[2]
Grade 2
[3]
Grade 4
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Anne Chang, MD
Organization: Stanford University
Phone: (650) 721 7151
Responsible Party: Anne Chang, Stanford University
ClinicalTrials.gov Identifier: NCT01529450     History of Changes
Other Study ID Numbers: SKIN0009
SU-09022011-8371 ( Other Identifier: Stanford University )
21759 ( Other Identifier: Stanford IRB )
First Submitted: October 11, 2011
First Posted: February 8, 2012
Results First Submitted: March 11, 2016
Results First Posted: November 2, 2016
Last Update Posted: January 16, 2017