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Trial record 4 of 32 for:    "Nevoid basal cell carcinoma syndrome"

Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients

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ClinicalTrials.gov Identifier: NCT02762084
Recruitment Status : Completed
First Posted : May 4, 2016
Results First Posted : July 15, 2019
Last Update Posted : July 15, 2019
Sponsor:
Information provided by (Responsible Party):
PellePharm, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Basal Cell Nevus Syndrome
Interventions Drug: Patidegib
Drug: Vehicle gel
Enrollment 17
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks
Period Title: Overall Study
Started 6 6 5
Received at Least 1 Dose of Study Drug 6 6 5
By Tumor Per Protocol Population (PP) [1] 6 6 4
Completed 5 6 4
Not Completed 1 0 1
Reason Not Completed
Withdrawal by Subject             1             0             0
Adverse Event             0             0             1
[1]
All treatment-targeted tumors except those biopsied and those that did not meet protocol criteria.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel Total
Hide Arm/Group Description Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks Total of all reporting groups
Overall Number of Baseline Participants 6 6 5 17
Hide Baseline Analysis Population Description
Participants who received at least 1 dose of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 6 participants 5 participants 17 participants
60.7  (12.14) 62.5  (13.87) 58.8  (17.58) 60.8  (13.63)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 5 participants 17 participants
Female
5
  83.3%
3
  50.0%
3
  60.0%
11
  64.7%
Male
1
  16.7%
3
  50.0%
2
  40.0%
6
  35.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 5 participants 17 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
6
 100.0%
6
 100.0%
5
 100.0%
17
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 5 participants 17 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
6
 100.0%
6
 100.0%
5
 100.0%
17
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United Kingdom Number Analyzed 6 participants 6 participants 5 participants 17 participants
6 6 5 17
Weight   [1] 
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 6 participants 5 participants 5 participants 16 participants
90.73  (20.539) 81.60  (11.872) 81.52  (21.477) 85.00  (17.95)
[1]
Measure Analysis Population Description: Weight data at baseline was not available for 1 participant in the Patidegib Gel 4% treatment arm.
1.Primary Outcome
Title Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline
Hide Description SEBs were defined as clinically diagnosed basal cell carcinoma (BCC) 5 millimeters (mm) or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percent change in greatest diameters of treatment-targeted surgically eligible basal cell carcinomas (SEBs) from Baseline to Week 26 was calculated as follows: (sum [Baseline] - sum [Week 26] / sum [Baseline] * 100), where sum = the greatest diameters of Baseline treatment-targeted SEBs and positive numbers represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using Last-Observation Carried Forward (LOCF).
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
By tumor per protocol population includes all treatment-targeted tumors except tumors that were biopsied and excludes tumors that did not meet protocol criteria.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 4
Overall Number of Units Analyzed
Type of Units Analyzed: Number of tumors
21 24 16
Mean (Standard Deviation)
Unit of Measure: percentage change
51.29  (41.780) 26.63  (41.270) 21.82  (25.213)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Patidegib Gel 2%, Vehicle Gel
Comments at Week 26
Type of Statistical Test Other
Comments Pairwise comparison
Statistical Test of Hypothesis P-Value 0.030
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA with treatment group as a factor and Baseline value as a covariate
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Patidegib Gel 4%, Vehicle Gel
Comments at Week 26
Type of Statistical Test Other
Comments Pairwise comparison
Statistical Test of Hypothesis P-Value 0.756
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA with treatment group as a factor and Baseline value as a covariate
2.Primary Outcome
Title Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline
Hide Description SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9-mm or greater at sites other than the face. A single baseline SEB designated as a treatment targeted tumor at Baseline was biopsied first at Baseline and again following 6 weeks of treatment. This was used to assess percent change in GLI1 mRNA levels as follows: (Baseline - Week 6) / Baseline * 100, where positive numbers to represent decrease in GL1 mRNA level and negative numbers to represent increase in GL1 mRNA level. Any missing values were not imputed; all available data is summarized.
Time Frame Baseline, Week 6
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug who had evaluable GLI1 mRNA data at Baseline and Week 6.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 4 4 4
Mean (Standard Deviation)
Unit of Measure: percentage change
53.83  (27.197) 20.69  (34.730) 28.53  (43.096)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Patidegib Gel 2%, Vehicle Gel
Comments at Week 6
Type of Statistical Test Other
Comments Pairwise comparison
Statistical Test of Hypothesis P-Value 0.500
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA with treatment group as a factor and using Baseline value as a covariate
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Patidegib Gel 4%, Vehicle Gel
Comments at Week 6
Type of Statistical Test Other
Comments Pairwise comparison
Statistical Test of Hypothesis P-Value 0.681
Comments [Not Specified]
Method ANCOVA
Comments ANCOVA with treatment group as a factor and using Baseline value as a covariate
3.Primary Outcome
Title Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Treatment-emergent Adverse Events Causally Related to Study Drug.
Hide Description All serious adverse events (SAEs) and all other non-serious adverse events (AEs) regardless of causality are located in the Reported AE Module. The number of AEs considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug.
Time Frame Baseline through Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants who were randomized, received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety assessment.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 5
Measure Type: Number
Unit of Measure: events
2 7 5
4.Primary Outcome
Title Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With Treatment-emergent Administrative Site Skin Condition AEs Causally Related to Study Drug.
Hide Description All SAEs and all other non-serious AEs, regardless of causality, are located in the Reported AE Module. The number of participants reporting administrative-site, skin condition treatment-emergent AEs considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug.
Time Frame Baseline through Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled participants who were randomized, received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety assessment.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 5
Measure Type: Number
Unit of Measure: participants
Application site alopecia 0 1 0
Application site dermatitis 0 1 0
Application site pain 0 1 0
Application site rash 0 1 0
Application site reaction 0 0 1
5.Secondary Outcome
Title The Number of Participants Reporting New SEBs on the Face From Baseline for the Combined Patidegib Treatment Groups
Hide Description Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. New facial SEBs are defined as those located at a site where there was no visible BCC of any size at Baseline. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug.
Arm/Group Title Patidegib Gel Vehicle Gel
Hide Arm/Group Description:
Patidegib: 2% or 4% gel applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 12 5
Measure Type: Number
Unit of Measure: participants
2 3
6.Secondary Outcome
Title The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups
Hide Description Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. New facial SEBs are defined as those located at a site where there was no visible BCC of any size at Baseline. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF. The mean number of new SEBs (number per participant) are presented. No measure of dispersion/precision was calculated.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug.
Arm/Group Title Patidegib Gel Vehicle Gel
Hide Arm/Group Description:
Patidegib: 2% or 4% gel applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 12 5
Mean (Standard Deviation)
Unit of Measure: new SEBs
0.4  (0.8) 1.4  (1.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Patidegib Gel, Vehicle Gel
Comments at Week 26
Type of Statistical Test Other
Comments One-sided comparison
Statistical Test of Hypothesis P-Value 0.048
Comments [Not Specified]
Method Mann Whitey U
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Patidegib Gel, Vehicle Gel
Comments at Week 26
Type of Statistical Test Other
Comments Two-sided comparison
Statistical Test of Hypothesis P-Value 0.096
Comments [Not Specified]
Method Mann Whitey U
Comments [Not Specified]
7.Secondary Outcome
Title The Mean Number of New SEBs on the Face for the Combined Patidegib Treatment Groups by Tumor Population
Hide Description Facial SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin. New facial SEBs are defined as those located at a site where there was no visible BCC of any size at Baseline. New facial SEBs were investigated for participants on vehicle gel versus participants on patidegib 2% and 4% gel. Missing values were imputed using LOCF. The mean number of new SEBs (number per participant) are presented. No measure of dispersion/precision was calculated.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
By tumor per protocol population includes all treatment-targeted tumors except tumors that were biopsied and excludes tumors that did not meet protocol criteria.
Arm/Group Title Patidegib Gel Vehicle Gel
Hide Arm/Group Description:
Patidegib: 2% or 4% gel applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 12 5
Mean (Standard Deviation)
Unit of Measure: new SEBs
0.3  (0.8) 1.4  (1.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Patidegib Gel, Vehicle Gel
Comments at Week 26
Type of Statistical Test Other
Comments Two-sided comparison
Statistical Test of Hypothesis P-Value 0.008
Comments [Not Specified]
Method Mann Whitey U
Comments [Not Specified]
8.Secondary Outcome
Title Percent Change in Baseline Treatment-targeted SEBs Tumor Size From Baseline
Hide Description SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percent change in greatest diameters of Baseline treatment-targeted SEBs from Baseline to Week x (Week 6, 10, 14, 18, or 22) was calculated as follows: (sum [Baseline] - sum [Week x] / sum [Baseline] * 100), where sum = the greatest diameters of Baseline treatment-targeted SEBs, and positive numbers to represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using LOCF.
Time Frame Baseline and Weeks 6, 10, 14, 18, and 22
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 5
Mean (Standard Deviation)
Unit of Measure: percentage change
Week 6 16.0  (37.07) 6.1  (10.41) 8.2  (4.55)
Week 10 30.8  (39.54) 10.7  (12.00) 10.5  (5.35)
Week 14 36.1  (46.45) 14.1  (13.18) 10.7  (6.26)
Week 18 26.0  (73.79) 18.4  (26.86) 19.6  (21.98)
Week 22 32.1  (78.71) 23.2  (22.87) 23.2  (28.03)
9.Secondary Outcome
Title Percent Change in Central Facial SEBs From Baseline
Hide Description Central facial SEBs were defined as those located on the nose or periorbital area (eyelids) which were 3 mm or greater at Baseline. The percent change from Baseline to Week x (Week x = Weeks 6, 10, 14, 18, 22, or 26) in central facial SEBs was calculated as follows: [sum (Baseline) - sum (Week x)] / [sum (Baseline)] * 100 where sum = the greatest diameters of Baseline treatment-targeted SEBs where positive numbers to represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using LOCF.
Time Frame Baseline and Weeks 6, 10, 14, 18, 22, and 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug and who had a central facial SEB at Baseline.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 1 2 2
Mean (Standard Deviation)
Unit of Measure: percent change
Week 6 5.9 [1]   (NA) -20.0  (28.28) 8.0  (24.11)
Week 10 5.9 [1]   (NA) -10.0  (42.43) 12.5  (17.68)
Week 14 5.9 [1]   (NA) -60.0  (84.85) 29.5  (28.93)
Week 18 5.9 [1]   (NA) -70.0  (70.71) 17.0  (11.25)
Week 22 5.9 [1]   (NA) -45.0  (63.64) 29.5  (28.93)
Week 26 5.9 [1]   (NA) -65.0  (63.64) 20.5  (41.78)
[1]
Standard deviation was not calculated because there was only 1 participant in the analysis population.
10.Secondary Outcome
Title Proportion of Non-central Facial BCCs Increasing to ≥ 5 mm From Baseline
Hide Description The proportion of non-central facial BCCs that at Baseline measured a greatest diameter of < 5 mm and increased to a diameter of ≥ 5 mm by Week x (Week x = Weeks 6, 10, 14, 18, 22, or 26) were calculated for each participant as follows: (Number of non−central facial BCCs with greatest diameter ≥ 5 mm at Week x) / (Number of non−central facial BCCs with greatest diameter < 5 mm at Baseline). Missing values were imputed using LOCF.
Time Frame Baseline and Weeks 6, 10, 14, 18, 22, and 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug with non-central facial BCCs < 5 mm at Baseline.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 3 2 3
Mean (Standard Deviation)
Unit of Measure: proportion of BCCs
Week 6 0.17  (0.289) 0  (0) 0.17  (0.289)
Week 10 0  (0) 0.67  (0.474) 0.17  (0.289)
Week 14 0  (0) 0  (0) 0  (0)
Week 18 0  (0) 0.17  (0.233) 0  (0)
Week 22 0  (0) 0.34  (0.474) 0  (0)
Week 26 0  (0) 0  (0) 0.11  (0.191)
11.Secondary Outcome
Title Proportion of Treatment-Targeted SEBs No Longer Classified as SEBs After 26 Weeks
Hide Description

SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The proportion of Baseline treatment-targeted SEBs that at the end of 26 weeks of treatment were no longer large enough to be classified as SEBs (that is, the proportion of Baseline treatment targeted SEBs on the face that became < 5 mm in greatest diameter and non-facial Baseline treatment targeted SEBs that became < 9 mm in greatest diameter) were calculated for each participant as follows:

(Number of Baseline treatment-targeted facial SEBs with greatest diameter < 5 mm) + (Baseline treatment targeted non-facial SEBs with greatest diameter < 9 mm) / Number of baseline treatment targeted SEBs.

Missing values were imputed using LOCF.

Time Frame Baseline and Weeks 6, 10, 14, 18, 22, and 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 5
Mean (Standard Deviation)
Unit of Measure: proportion of SEBs
Week 6 0.37  (0.497) 0.13  (0.242) 0.12  (0.179)
Week 10 0.40  (0.473) 0.13  (0.103) 0.16  (0.219)
Week 14 0.53  (0.468) 0.23  (0.234) 0.16  (0.219)
Week 18 0.57  (0.497) 0.23  (0.320) 0.28  (0.303)
Week 22 0.53  (0.516) 0.30  (0.276) 0.32  (0.363)
Week 26 0.53  (0.516) 0.30  (0.303) 0.36  (0.434)
12.Other Pre-specified Outcome
Title Percentage of Treatment-targeted SEBs Achieving Clear or Almost Clear on the 5-point Investigator Static Global Tumor Assessment (ISGTA) Scale
Hide Description The ISGTA is a scale with scores ranging from 0 (clear), 1 (almost clear), 2 (minimal residual tumor), to 3 (clearly visible tumor). The Investigator assessed each Baseline treatment-targeted SEB at Weeks 6, 10, 14, 18, 22, and 26. SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percentage of Baseline treatment-targeted SEBs evaluated as being clear or almost clear at Week x (Week x = Week 6, 10, 14, 18, 22 or 26) based on the ISGTA scale was calculated as follows: (Number of baseline treatment-targeted SEBs with ISGTA score of 0 or 1 at Week x) / (Number of Baseline treatment-targeted SEBs) * 100. Missing data were imputed using LOCF. The percentage of responders achieving clear (0) or almost clear (1) on the ISGTA scale are presented by Week.
Time Frame Baseline and Weeks 6, 10, 14, 18, 22, and 26
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who received at least 1 dose of study drug.
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description:
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Applied topically twice daily for 26 weeks
Overall Number of Participants Analyzed 6 6 5
Measure Type: Number
Unit of Measure: percentage of SEBs
Week 6 23.3 3.3 12.0
Week 10 23.3 13.3 8.0
Week 14 33.3 13.3 8.0
Week 18 33.3 23.3 20.0
Week 22 36.7 26.7 20.0
Week 26 33.3 30.0 24.0
Time Frame 26 Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Hide Arm/Group Description Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks Applied topically twice daily for 26 weeks
All-Cause Mortality
Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)      0/6 (0.00%)      0/5 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/6 (0.00%)      0/6 (0.00%)      2/5 (40.00%)    
Infections and infestations       
Pneumonia pneumococcal * 1  0/6 (0.00%)  0 0/6 (0.00%)  0 1/5 (20.00%)  1
Pneumonia * 1  0/6 (0.00%)  0 0/6 (0.00%)  0 1/5 (20.00%)  1
1
Term from vocabulary, MedDRA (19.0)
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Patidegib Gel 2% Patidegib Gel 4% Vehicle Gel
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/6 (66.67%)      6/6 (100.00%)      3/5 (60.00%)    
Endocrine disorders       
Hyperthyroidism * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Hypothyroidism * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Eye disorders       
Episcleritis * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Gastrointestinal disorders       
Constipation * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Diarrhoea * 1  1/6 (16.67%)  2/6 (33.33%)  0/5 (0.00%) 
Food Poisioning * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Nausea * 1  0/6 (0.00%)  1/6 (16.67%)  1/5 (20.00%) 
General disorders       
Application site alopecia * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Application site dermatitis * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Application site pain * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Application site rash * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Application site reaction * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Fatigue * 1  2/6 (33.33%)  3/6 (50.00%)  2/5 (40.00%) 
Infections and infestations       
Candida infection * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Cellulitis * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Nasopharyngitis * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Oral herpes * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Viral upper respiratory tract infection * 1  1/6 (16.67%)  1/6 (16.67%)  0/5 (0.00%) 
Metabolism and nutrition disorders       
Diabetes mellitus * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Hypercholesterolaemina * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Musculoskeletal and connective tissue disorders       
Arthritis * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Muscle spasm * 1  1/6 (16.67%)  1/6 (16.67%)  2/5 (40.00%) 
Nervous system disorders       
Ageusia * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Dysgeusia * 1  0/6 (0.00%)  1/6 (16.67%)  1/5 (20.00%) 
Trigeminal neuralgia * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Psychiatric disorders       
Depressed mood * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Renal and urinary disorders       
Acute kidney injury * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Nephrolithiasis * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough * 1  1/6 (16.67%)  0/6 (0.00%)  1/5 (20.00%) 
Dysphonia * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Skin and subcutaneous tissue disorders       
Alopecia * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Hair growth abnormal * 1  1/6 (16.67%)  0/6 (0.00%)  0/5 (0.00%) 
Pruritus * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
Rash * 1  0/6 (0.00%)  1/6 (16.67%)  0/5 (0.00%) 
Vascular disorders       
Hypertension * 1  0/6 (0.00%)  0/6 (0.00%)  1/5 (20.00%) 
1
Term from vocabulary, MedDRA (19.0)
*
Indicates events were collected by non-systematic assessment
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
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: PellePharm, Inc.
Phone: (510) 502-6144
EMail: Info@Pellepharm.com
Layout table for additonal information
Responsible Party: PellePharm, Inc.
ClinicalTrials.gov Identifier: NCT02762084     History of Changes
Other Study ID Numbers: Pelle-926-201
2015-004274-15 ( EudraCT Number )
First Submitted: April 27, 2016
First Posted: May 4, 2016
Results First Submitted: June 28, 2018
Results First Posted: July 15, 2019
Last Update Posted: July 15, 2019