Trial Outcomes & Findings for Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome) (NCT NCT04308395)

Last Updated: 2024-09-24

Results Overview

IMP=Investigational medicinal product; Unique adverse event = adverse event of a certain preferred term, counted only once within each subject. Related adverse event = adverse event with relationship as Definitely, Probably or Possibly.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

108 participants

Primary outcome timeframe

12 months

Results posted on

2024-09-24

Participant Flow

Participant milestones

Participant milestones
Measure	Patidegib Topical Gel, 2% Patidegib Topical Gel, 2% Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
Overall Study STARTED	108
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	105

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

2 participants missing

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patidegib Topical Gel, 2%, n=105 Participants All study subjects were treated with Patidegib Topical Gel 2% (the investigational medicinal product \[IMP\]), applied topically twice daily to the face.
Age, Continuous	53.9 years STANDARD_DEVIATION 13.9 • n=105 Participants
Sex: Female, Male Female	59 Participants n=105 Participants
Sex: Female, Male Male	46 Participants n=105 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=105 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	84 Participants n=105 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	15 Participants n=105 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=105 Participants
Race (NIH/OMB) Asian	0 Participants n=105 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	8 Participants n=105 Participants
Race (NIH/OMB) Black or African American	0 Participants n=105 Participants
Race (NIH/OMB) White	87 Participants n=105 Participants
Race (NIH/OMB) More than one race	2 Participants n=105 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants n=105 Participants
Height	175.9 cm STANDARD_DEVIATION 10.0 • n=103 Participants • 2 participants missing
Weight	93.1 kg STANDARD_DEVIATION 21.4 • n=105 Participants
BMI	29.9 kg/m2 STANDARD_DEVIATION 6.2 • n=103 Participants • 2 participants missing

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Patidegib Topical Gel, 2% n=105 Participants Patidegib Topical Gel, 2% Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2%
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one adverse event related to IMP leading to death	0 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one adverse event	46 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one adverse event related to IMP	6 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least leading to treatment discontinuation	0 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one AE related to IMP leading to treatment discontinuation	0 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one paralesional adverse event	3 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one serious adverse event	3 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects with at least one serious adverse event related to IMP	0 Number of subjects
Incidence of Treatment-emergent Adverse Events (TEAEs) Number of subjects withat least one adverse event leading to death	0 Number of subjects

PRIMARY outcome

Timeframe: 12 months