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Study was terminated early due to low blinded event rate and this study termination is not related to safety of the drug Patidegib Topical Gel, 2%.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patidegib Topical Gel, 2% | Experimental | Patidegib Topical Gel, 2% |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Patidegib Topical Gel, 2% | Drug | Patidegib Topical Gel, 2% |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
| Measure | Description | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director VP, Clinical Operations | PellePharm, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| PellePharm Investigative Site | Fremont | California | 94538 | United States | ||
| PellePharm Investigative Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | Patidegib Topical Gel, 2% | Patidegib Topical Gel, 2% Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2% |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 30, 2020 | Aug 26, 2024 |
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All patients will receive Patidegib Topical Gel, 2%,
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| 12 months |
| Change in Dermatology Life Quality Index (DLQI) | 12 months |
| Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L) | 12 months |
| Newport Beach |
| California |
| 92660 |
| United States |
| Yale University | New Haven | Connecticut | 06519 | United States |
| Pellepharm Investigative Site | Miami | Florida | 33125 | United States |
| PellePharm Investigative Site | Ormond Beach | Florida | 32174 | United States |
| PellePharm Investigative Site | Chicago | Illinois | 60637 | United States |
| Laser & Skin Surgery Center of Indiana | Indianapolis | Indiana | 46260 | United States |
| Pellepharm Investigative Site | Ann Arbor | Michigan | 48109 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Saint Louis University | St Louis | Missouri | 63104 | United States |
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Penn State Health Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| University of Utah, Midvalley Dermatology | Murray | Utah | 84107 | United States |
| PellePhram Investigative Site | Leuven | 3000 | Belgium |
| PellePharm Investigative Site | Copenhagen | DK-2400 | Denmark |
| PellePharm Investigative Site | Lille | France |
| PellePharm Investigative Site | Nantes | 44093 | France |
| Hopital Saint Louis | Paris | 75010 | France |
| PellePharm Investigative Site | Berlin | 10117 | Germany |
| PellePharm Investigative Site | Munich | 80337 | Germany |
| PellePharm Investigative Site | Münster | 48149 | Germany |
| AOU Luigi Vanvitelli | Naples | 80131 | Italy |
| PellePharm Investigative Site | Rozzano | 20089 | Italy |
| PellePharm Investigative Site | Vicenza | 36100 | Italy |
| PellePharm Investigative Site | Maastricht | 6229 HX | Netherlands |
| PellePharm Investigative Site | Barcelona | 08036 | Spain |
| PellePharm Investigative Site | Madrid | 28023 | Spain |
| PellePharm Investigative Site | Madrid | 28034 | Spain |
| PellePharm Investigative Site | Seville | 41009 | Spain |
| PellePharm Investigative Site | Glasgow | United Kingdom |
| PellePharm Investigative Site | London | United Kingdom |
| PellePharm Investigative Site | Manchester | United Kingdom |
| PellePharm Investigative Site | Oxford | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Height | 2 participants missing | Mean | Standard Deviation | cm |
| ||||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||||
| BMI | 2 participants missing | Mean | Standard Deviation | kg/m2 |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Number | Number of subjects | 12 months |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change in Number of Facial BCCs Removed by Surgery | Due to the Sponsor's decision to discontinue the trial, no secondary outcomes data was collected | Posted | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Number of New Lesions Suspicious for BCC | Due to the Sponsor's decision to discontinue the trial, no secondary outcomes data was collected | Posted | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Score | Due to the Sponsor's decision to discontinue the trial, no secondary outcomes data was collected | Posted | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Dermatology Life Quality Index (DLQI) | Due to the Sponsor's decision to discontinue the trial, no secondary outcomes data was collected | Posted | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Change in EuroQol Group 5-level EQ 5D (EQ 5D 5L) | Due to the Sponsor's decision to discontinue the trial, no secondary outcomes data was collected | Posted | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||
| 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. | Posted | Number | Number of adverse events | 12 months |
|
|
1 Year
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Patidegib Topical Gel, 2% | Patidegib Topical Gel, 2% Patidegib Topical Gel, 2%: Patidegib Topical Gel, 2% | 0 | 105 | 3 | 105 | 46 | 105 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | Non-systematic Assessment |
| ||
| Craniocerebral injury | Injury, poisoning and procedural complications | Non-systematic Assessment |
| ||
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| No adverse event occured in 5% or more of the patients | General disorders | Non-systematic Assessment | No adverse event occured in 5% or more of the patients |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| VP Clinical & Regulatory Affairs | Sol-Gel Technologies, Ltd. | +972-73-3729453 | Ofra.Levy-Hacham@sol-gel.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 16, 2020 | Aug 26, 2024 | SAP_003.pdf |
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| ID | Term |
|---|---|
| D001478 | Basal Cell Nevus Syndrome |
| D002280 | Carcinoma, Basal Cell |
| ID | Term |
|---|---|
| D009807 | Odontogenic Cysts |
| D007570 | Jaw Cysts |
| D001845 | Bone Cysts |
| D003560 | Cysts |
| D009369 | Neoplasms |
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D018295 | Neoplasms, Basal Cell |
| D009386 | Neoplastic Syndromes, Hereditary |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D007571 | Jaw Diseases |
| D009057 | Stomatognathic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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|
| Unknown or Not Reported |
|
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| Number of subjects with at least one AE related to IMP leading to treatment discontinuation |
|
| Number of subjects with at least one paralesional adverse event |
|
| Number of subjects with at least one serious adverse event |
|
| Number of subjects with at least one serious adverse event related to IMP |
|
| Number of subjects withat least one adverse event leading to death |
|
| Number of subjects with at least one adverse event related to IMP leading to death |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Total number of adverse events |
| |||||
| Number of unique adverse events |
|