Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

Status

Terminated

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04308395

Enrollment

108

Registered

2020-03-16

Start date

2020-06-03

Completion date

2021-07-14

Last updated

2024-09-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Basal Cell Nevus Syndrome

Keywords

Gorlin syndrome, Patidegib, Basal cell nevus syndrome, Nevoid basal cell carcinoma syndrome, Basal cell carcinoma, Hedgehog, Surgically eligible basal cell carcinomas, BCC, BCNS

Brief summary

This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.

Interventions

DRUGPatidegib Topical Gel, 2%

Patidegib Topical Gel, 2%

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

All patients will receive Patidegib Topical Gel, 2%,

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. The subject must have completed PellePharm Study Pelle-926-201 or Pelle-926-301. 2. Study Pelle-926-301 subjects must have completed the End of Treatment Visit in Study 301, prior to the Screening Visit in this study. They must also complete all Study 301 related procedures prior to the Baseline Visit of this study. 3. The subject must be willing to abstain from application of a non-study topical medication (prescription or over the counter) to facial skin for the duration of the trial except as prescribed by the Investigator. Moisturizers and emollients are allowed. Subjects will be encouraged to use their preferred sunscreen with a sunscreen protection factor (SPF) of at least 30 daily on all exposed skin sites. 4. Female subjects must have a negative pregnancy test. For Study 301 subjects a negative serum pregnancy test result from Study 301 is acceptable if the test was done within 7 days of the Screening Visit of this study. 5. If the subject is a woman of child bearing potential (WOCBP), she must be willing to use birth control methods which may be considered highly effective. Hormonal contraception must be supplemented with a barrier method (preferably condom). Birth control must start prior to Baseline, continue through the duration of the study, and for 30 days after last application of investigational product (IP). 6. If the subject is a male with a female sex partner who is a WOCBP, the subject must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 3 months after the last application of IP. 7. The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator. 8. The subject is willing to forego treatment of facial BCCs with anything other than the study IP except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. In such instances, the only other allowed form of treatment is surgical.

Exclusion criteria

1. The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP. 2. The subject has current, recent (within five half lives of the experimental drug or if half life not known, within the past 6 months prior to the Screening Visit), or planned (while enrolled in this study) participation in an experimental drug study (excluding Study 301). 3. The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures. 4. The subject is pregnant or breastfeeding.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of Treatment-emergent Adverse Events (TEAEs)	12 months	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.

Secondary

Measure	Time frame
Change in Number of Facial BCCs Removed by Surgery	12 months
Change in Number of New Lesions Suspicious for BCC	12 months
Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Score	12 months
Change in Dermatology Life Quality Index (DLQI)	12 months
Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L)	12 months

Countries

Belgium, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States

Participant flow

Participants by arm

Arm	Count
Patidegib Topical Gel, 2%, All study subjects were treated with Patidegib Topical Gel 2% (the investigational medicinal product \[IMP\]), applied topically twice daily to the face.	105
Total	105

Baseline characteristics

Characteristic	Patidegib Topical Gel, 2%,
Age, Continuous	53.9 years STANDARD_DEVIATION 13.9
BMI	29.9 kg/m2 STANDARD_DEVIATION 6.2
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	84 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	15 Participants
Height	175.9 cm STANDARD_DEVIATION 10
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	2 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	8 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants
Race (NIH/OMB) White	87 Participants
Sex: Female, Male Female	59 Participants
Sex: Female, Male Male	46 Participants
Weight	93.1 kg STANDARD_DEVIATION 21.4

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 105
other Total, other adverse events	46 / 105
serious Total, serious adverse events	3 / 105

Outcome results

Primary

Incidence of Treatment-emergent Adverse Events (TEAEs)

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.

Time frame: 12 months

Arm	Measure	Group	Value (NUMBER)
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least one serious adverse event	3 Number of subjects
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least one adverse event	46 Number of subjects
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least one adverse event related to IMP	6 Number of subjects
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least leading to treatment discontinuation	0 Number of subjects
Patidegib Topical Gel, 2%	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
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least one paralesional adverse event	3 Number of subjects
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects with at least one serious adverse event related to IMP	0 Number of subjects
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of subjects withat least one adverse event leading to death	0 Number of subjects
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

Primary

Incidence of Treatment-emergent Adverse Events (TEAEs)

Time frame: 12 months

Arm	Measure	Group	Value (NUMBER)
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Total number of adverse events	82 Number of adverse events
Patidegib Topical Gel, 2%	Incidence of Treatment-emergent Adverse Events (TEAEs)	Number of unique adverse events	72 Number of adverse events

Secondary