Clinical trial NCT04308395: Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
Official title: A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
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
Lead sponsor: Sol-Gel Technologies, Ltd.
Sponsor class: INDUSTRY
First posted: 2020-03-16
Enrollment: 108
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: All patients will receive Patidegib Topical Gel, 2%,
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of Treatment-emergent Adverse Events (TEAEs)
timeFrame: 12 months
Secondary outcomes:
measure: Change in Number of Facial BCCs Removed by Surgery
timeFrame: 12 months
measure: Change in Number of New Lesions Suspicious for BCC
timeFrame: 12 months
measure: Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Score
timeFrame: 12 months
measure: Change in Dermatology Life Quality Index (DLQI)
timeFrame: 12 months
measure: Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L)
timeFrame: 12 months
Other outcomes:
Locations:
city: Fremont
country: United States
facility: PellePharm Investigative Site
state: California
city: Newport Beach
country: United States
facility: PellePharm Investigative Site
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Miami
country: United States
facility: Pellepharm Investigative Site
state: Florida
city: Ormond Beach
country: United States
facility: PellePharm Investigative Site
state: Florida
city: Chicago
country: United States
facility: PellePharm Investigative Site
state: Illinois
city: Indianapolis
country: United States
facility: Laser & Skin Surgery Center of Indiana
state: Indiana
city: Ann Arbor
country: United States
facility: Pellepharm Investigative Site
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Saint Louis University
state: Missouri
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Murray
country: United States
facility: University of Utah, Midvalley Dermatology
state: Utah
city: Leuven
country: Belgium
facility: PellePhram Investigative Site
city: Copenhagen
country: Denmark
facility: PellePharm Investigative Site
city: Lille
country: France
facility: PellePharm Investigative Site
city: Nantes
country: France
facility: PellePharm Investigative Site
city: Paris
country: France
facility: Hopital Saint Louis
city: Berlin
country: Germany
facility: PellePharm Investigative Site
city: Munich
country: Germany
facility: PellePharm Investigative Site
city: Münster
country: Germany
facility: PellePharm Investigative Site
city: Napoli
country: Italy
facility: AOU Luigi Vanvitelli
city: Rozzano
country: Italy
facility: PellePharm Investigative Site
city: Vicenza
country: Italy
facility: PellePharm Investigative Site
city: Maastricht
country: Netherlands
facility: PellePharm Investigative Site
city: Barcelona
country: Spain
facility: PellePharm Investigative Site
city: Madrid
country: Spain
facility: PellePharm Investigative Site
city: Madrid
country: Spain
facility: PellePharm Investigative Site
city: Sevilla
country: Spain
facility: PellePharm Investigative Site
city: Glasgow
country: United Kingdom
facility: PellePharm Investigative Site
city: London
country: United Kingdom
facility: PellePharm Investigative Site
city: Manchester
country: United Kingdom
facility: PellePharm Investigative Site
city: Oxford
country: United Kingdom
facility: PellePharm Investigative Site
Study officials:
affiliation: PellePharm, Inc.
name: Study Director VP, Clinical Operations
role: STUDY_DIRECTOR
References: