Trial Outcomes & Findings for Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome) (NCT NCT03703310)
NCT ID: NCT03703310
Last Updated: 2024-07-03
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
174 participants
Primary outcome timeframe
Month 12
Results posted on
2024-07-03
Participant Flow
Participant milestones
| Measure |
Patidegib Topical Gel, 2%,
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Overall Study
STARTED
|
87
|
87
|
|
Overall Study
COMPLETED
|
67
|
70
|
|
Overall Study
NOT COMPLETED
|
20
|
17
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Some countries do not allow race and/or ethnicity collection
Baseline characteristics by cohort
| Measure |
Patidegib Topical Gel, 2%,
n=87 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=87 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
Total
n=174 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
51.9 years
STANDARD_DEVIATION 15.45 • n=87 Participants
|
51.8 years
STANDARD_DEVIATION 13.40 • n=87 Participants
|
51.8 years
STANDARD_DEVIATION 14.42 • n=174 Participants
|
|
Sex: Female, Male
Female
|
48 Participants
n=87 Participants
|
49 Participants
n=87 Participants
|
97 Participants
n=174 Participants
|
|
Sex: Female, Male
Male
|
39 Participants
n=87 Participants
|
38 Participants
n=87 Participants
|
77 Participants
n=174 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=81 Participants • Some countries do not allow race and/or ethnicity collection
|
6 Participants
n=83 Participants • Some countries do not allow race and/or ethnicity collection
|
8 Participants
n=164 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
76 Participants
n=81 Participants • Some countries do not allow race and/or ethnicity collection
|
75 Participants
n=83 Participants • Some countries do not allow race and/or ethnicity collection
|
151 Participants
n=164 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=81 Participants • Some countries do not allow race and/or ethnicity collection
|
2 Participants
n=83 Participants • Some countries do not allow race and/or ethnicity collection
|
5 Participants
n=164 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
2 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
2 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
0 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
White
|
79 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
78 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
157 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
1 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
2 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=82 Participants • Some countries do not allow race and/or ethnicity collection
|
3 Participants
n=84 Participants • Some countries do not allow race and/or ethnicity collection
|
5 Participants
n=166 Participants • Some countries do not allow race and/or ethnicity collection
|
|
Height
|
176.30 cm
STANDARD_DEVIATION 9.390 • n=87 Participants
|
176.07 cm
STANDARD_DEVIATION 10.421 • n=87 Participants
|
176.18 cm
STANDARD_DEVIATION 9.894 • n=174 Participants
|
|
Weight
|
93.06 kg
STANDARD_DEVIATION 22.812 • n=87 Participants
|
91.75 kg
STANDARD_DEVIATION 20.101 • n=87 Participants
|
92.41 kg
STANDARD_DEVIATION 21.447 • n=174 Participants
|
|
BMI
|
29.87 kg/m2
STANDARD_DEVIATION 6.062 • n=87 Participants
|
29.58 kg/m2
STANDARD_DEVIATION 5.845 • n=87 Participants
|
29.72 kg/m2
STANDARD_DEVIATION 5.938 • n=174 Participants
|
|
Time since Diagnosis of BCNS (basal cell nevus syndrome)
|
28.24 years
STANDARD_DEVIATION 16.960 • n=87 Participants
|
25.82 years
STANDARD_DEVIATION 16.379 • n=87 Participants
|
27.03 years
STANDARD_DEVIATION 16.668 • n=174 Participants
|
|
Number of SEBs (surgically eligible basal cell carcinoma) at Baseline
|
4.4 Numberof SEBs
STANDARD_DEVIATION 6.25 • n=87 Participants
|
5.0 Numberof SEBs
STANDARD_DEVIATION 6.68 • n=87 Participants
|
4.7 Numberof SEBs
STANDARD_DEVIATION 6.45 • n=174 Participants
|
|
Number of BCCs including SEBs at Baseline
|
13.9 Number of BCCs
STANDARD_DEVIATION 11.20 • n=87 Participants
|
15.4 Number of BCCs
STANDARD_DEVIATION 11.89 • n=87 Participants
|
14.7 Number of BCCs
STANDARD_DEVIATION 11.54 • n=174 Participants
|
|
Number of BCCs excluding SEBs at Baseline
|
9.5 Number of BCCs
STANDARD_DEVIATION 8.52 • n=87 Participants
|
10.4 Number of BCCs
STANDARD_DEVIATION 8.85 • n=87 Participants
|
9.9 Number of BCCs
STANDARD_DEVIATION 8.67 • n=174 Participants
|
|
Previous Hedgehog Inhibitor therapy
Yes
|
35 participants
n=87 Participants
|
33 participants
n=87 Participants
|
68 participants
n=174 Participants
|
|
Previous Hedgehog Inhibitor therapy
No
|
52 participants
n=87 Participants
|
54 participants
n=87 Participants
|
106 participants
n=174 Participants
|
PRIMARY outcome
Timeframe: Month 12Population: The number of BCCs per subject by Month 12 summarized subjects with BCC assessments within the Month 12 analysis window.
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=58 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=65 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Number of New BCCs Per Participant
|
2.84 New basal cell carcinomas
Standard Deviation 3.39
|
4.03 New basal cell carcinomas
Standard Deviation 4.76
|
SECONDARY outcome
Timeframe: Month 12Population: The number of nSEBs per subject by Month 12 summarizes subjects with BCC assessments within the Month 12 analysis window.
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=58 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=65 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Number of New Surgically Eligible BCCs (nSEBs) Per Participant
|
0.71 New basal cell carcinomas
Standard Deviation 1.28
|
0.91 New basal cell carcinomas
Standard Deviation 1.61
|
SECONDARY outcome
Timeframe: Month 12Population: subjects with no postbaseline data were excluded from the analysis
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=83 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=86 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Percentage of Participants Developing >=2 Facial New BCCs
|
45 Participants
|
55 Participants
|
SECONDARY outcome
Timeframe: Month 12Population: subjects with no postbaseline data were excluded from the analysis
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=83 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=86 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Percentage of Participants Developing >=1 Facial New BCCs
|
56 Participants
|
65 Participants
|
SECONDARY outcome
Timeframe: Month 9Population: subjects with no postbaseline data were excluded from the analysis
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=68 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=65 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Number of New BCCs Per Participant
|
2.46 New basal cell carcinomas
Standard Deviation 3.42
|
3.15 New basal cell carcinomas
Standard Deviation 3.94
|
SECONDARY outcome
Timeframe: Month 6Population: subjects with no postbaseline data were excluded from the analysis
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=68 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=69 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Number of New BCCs Per Participant
|
1.68 New basal cell carcinomas
Standard Deviation 2.82
|
2.12 New basal cell carcinomas
Standard Deviation 2.96
|
SECONDARY outcome
Timeframe: Month 12The Advanced Basal Cell Carcinoma Index (aBCCdex) measures the impact of basal cell carcinoma on patients' quality of life. The index consists of several subscales, each subscale with its own range of scores for each question Lesion Symptoms Scale: Scores range from 1 to 7 and it includes 6 questions. Therefore the total score range is from 6 (minimum) to 42 (maximum), where a higher score correlates with a poorer quality of life.
Outcome measures
| Measure |
Patidegib Topical Gel, 2%,
n=87 Participants
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=87 Participants
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Symptom Score Scale Score
|
11.49 score on a scale
Standard Deviation 18.365
|
15.54 score on a scale
Standard Deviation 21.002
|
Adverse Events
Patidegib Topical Gel, 2%,
Serious events: 5 serious events
Other events: 47 other events
Deaths: 0 deaths
Patidegib Topical Gel, Vehicle
Serious events: 7 serious events
Other events: 65 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Patidegib Topical Gel, 2%,
n=87 participants at risk
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=87 participants at risk
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Injury, poisoning and procedural complications
Concussion
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Injury, poisoning and procedural complications
Foot fracture
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Nervous system disorders
Brain injury
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Nervous system disorders
Cerebrovascular accident
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Infections and infestations
Vaginal abscess
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Infections and infestations
Erysipelas
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Infections and infestations
Pneumonia viral
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Infections and infestations
Post procedural sepsis
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Renal and urinary disorders
Acute kidney injury
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Vascular disorders
Shock haemorrhagic
|
1.1%
1/87 • Number of events 1 • 1 year
|
0.00%
0/87 • 1 year
|
|
Cardiac disorders
Angina pectoris
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Congenital, familial and genetic disorders
Morton's syndrome
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Eye disorders
Retinal detachment
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
|
Eye disorders
Retinal tear
|
0.00%
0/87 • 1 year
|
2.3%
2/87 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Inguinal hernia
|
0.00%
0/87 • 1 year
|
1.1%
1/87 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Patidegib Topical Gel, 2%,
n=87 participants at risk
Participants will be randomized to receive Patidegib Topical Gel, 2%. The Patidegib Topical Gel, 2% will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
|
Patidegib Topical Gel, Vehicle
n=87 participants at risk
Participants will be randomized to receive Vehicle. The Patidegib Topical Gel, Vehicle will be dispensed to participants at each study visit and applied topically twice daily to the face.
Patidegib Topical Gel, Vehicle: Patidegib Topical Gel, Vehicle
|
|---|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
5.7%
5/87 • 1 year
|
10.3%
9/87 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
5.7%
5/87 • 1 year
|
6.9%
6/87 • 1 year
|
|
Infections and infestations
Nasopharyngitis
|
2.3%
2/87 • 1 year
|
11.5%
10/87 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.7%
5/87 • 1 year
|
12.6%
11/87 • 1 year
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
4.6%
4/87 • 1 year
|
10.3%
9/87 • 1 year
|
|
General disorders
Fatigue
|
5.7%
5/87 • 1 year
|
4.6%
4/87 • 1 year
|
|
General disorders
Pyrexia
|
5.7%
5/87 • 1 year
|
1.1%
1/87 • 1 year
|
|
Nervous system disorders
Headache
|
5.7%
5/87 • 1 year
|
9.2%
8/87 • 1 year
|
|
Nervous system disorders
Paraesthesia
|
5.7%
5/87 • 1 year
|
3.4%
3/87 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.9%
6/87 • 1 year
|
4.6%
4/87 • 1 year
|
Additional Information
VP Clinical & Regulatory Affairs
Sol-Gel Technologies, Ltd.
Phone: +972-73-3729453
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place