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)

Recruitment Status
COMPLETED - HAS RESULTS
(See Contacts and Locations)Verified February 2021 by Sol-Gel Technologies, Ltd.
Sponsor
Sol-Gel Technologies, Ltd.
Information Provided by (Responsible Party)
Sol-Gel Technologies, Ltd.
Clinicaltrials.gov Identifier
NCT03703310
Other Study ID Numbers:
Pelle-926-301
First Submitted
October 8, 2018
First Posted
October 10, 2018
Results First Posted
May 28, 2024
Last Update Posted
July 2, 2024
Last Verified
February 2021

ClinicalTrials.gov processed this data on June 2024. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

An open-label, extension safety and tolerability study is planned for at least 12 months duration following the end of this study. All participants who complete the Month 12 Exit Visit having demonstrated adequate compliance with application of the Investigational Product (IP) without major Protocol Deviations (PDs) during the study will be eligible for participation in the extension study.

All participants will be contacted by phone approximately 30 days following the Exit or Discontinuation Visit to determine if the participant has experienced any new adverse events (AEs)/serious AEs (SAEs) since discontinuation/completion of study treatment.

Condition or DiseaseIntervention/Treatment
Basal Cell Nevus Syndrome
Drug: Patidegib Topical Gel, 2%Drug: Patidegib Topical Gel, Vehicle

Study Design

Study TypeInterventional
Actual Enrollment174 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingQuadruple
Primary PurposeTreatment
Official TitleA Multicenter, Randomized, Double-blind, Vehicle-controlled, Phase 3 Efficacy and Safety 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
Study Start DateFebruary 18, 2019
Actual Primary Completion DateDecember 27, 2020
Actual Study Completion DateDecember 27, 2020

Groups and Cohorts

Group/CohortIntervention/Treatment
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.
Drug: 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.
Drug: Patidegib Topical Gel, Vehicle
Patidegib Topical Gel, Vehicle

Outcome Measures

Primary Outcome Measures
  1. Number of New BCCs Per Participant
Secondary Outcome Measures
  1. Number of New Surgically Eligible BCCs (nSEBs) Per Participant
  2. Percentage of Participants Developing >=2 Facial New BCCs
  3. Percentage of Participants Developing >=1 Facial New BCCs
  4. Number of New BCCs Per Participant
  5. Number of New BCCs Per Participant
  6. Change in Advanced Basal Cell Carcinoma Index (aBCCdex) Lesion Symptom Score Scale Score
    The 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.

Eligibility Criteria

Ages Eligible for Study(Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
1. The participant must be age at least 18 years of age at the Screening Visit. 2. The participant must provide written informed consent prior to any study procedures. 3. The participant must meet diagnostic criteria for the basal cell nevus (Gorlin) syndrome including major criterion #3a plus 1 additional major criterion or plus 2 additional minor criteria listed below. Major criteria: 1. \>2 histologically confirmed BCCs or 1 for participant under age 20. 2. Odontogenic keratocysts of the jaw confirmed histologically. 3. ≥3 palmar and/or plantar pits seen at the Screening Visit. 4. Bilamellar calcification of the falx cerebri present at less than 20 years of age. 5. Fused, bifid, or markedly splayed ribs. 6. First degree relative with Gorlin syndrome. 7. Patched protein 1 (PTCH1) mutation predicted to be of functional significance in normal tissue. Minor criteria: 1. Macrocephaly. 2. Congenital malformations including frontal bossing, cleft lip or palate, "coarse face", moderate to severe hypertelorism. 3. Skeletal abnormalities detectable clinically: Sprengel deformity, marked pectus deformity, or marked finger syndactyly. 4. Skeletal abnormalities detectable radiographically: bridging of the sella turcica; vertebral abnormalities such as hemivertebrae, fusion or elongation of the vertebral bodies; modeling defects of the hands and feet; flame shaped lucencies of the hands or feet. 5. Ovarian fibroma. 6. Medulloblastoma (Modification of criteria of V Kimonis et al Am J Med Genet 69: 299-308, 1997). 4. The participant must have 10 (with at least 3 on the face) clinically typical BCCs present within 24 months prior to Randomization (Baseline/Day 1). Additionally, the subject must have at least 2 BCCs with longest diameter \<5 mm present on the face prior to Randomization (Baseline/Day 1). 5. The participant is willing to have blood collected to measure circulating drug levels. 6. The participant is 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. Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily on all exposed skin sites. 7. If the participant is a woman of childbearing potential (WOCBP), she must be willing to use complete abstinence from sexual intercourse and/or she and her partner must be willing to use at least 2 highly-effective forms of birth control starting prior to Baseline, through the duration of the study, and for 12 months after last application of IP. 8. If the participant is a male with a female sex partner who is a WOCBP, the participant must be willing to use condoms, even after a vasectomy, starting prior to Baseline, through the duration of the study, and for at least 8 months after the last application of IP. 9. The participant is willing for all facial BCCs to be evaluated and treatment recommendations made only by the Investigator. 10. The participant 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 facial BCC potentially might compromise the health of the subject. During the trial the only allowed form of treatment is surgical. Non-facial BCCs may be removed at the discretion of the Investigator or Primary Skin Care Physician (PSCP).
Exclusion Criteria
1. The subject has previously participated in a clinical trial evaluating patidegib topical gel. 2. The participant has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP. Among these are use of the following: 1. 5-fluorouracil, imiquimod, diclofenac, or Ingenol mebutate (except as topical treatment to discrete non-facial BCCs) systemically or topically to the skin within the 2 months prior to the Screening Visit. 2. Systemic chemotherapy within 1 year prior to the Screening Visit. 3. Known inhibitors of the Hedgehog signaling pathway (such as vismodegib, sonidegib, itraconazole) topically or systemically within 3 months prior to the Screening Visit. 4. Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 2 months prior to the Screening Visit. 3. The participant is known to have a hypersensitivity to any of the ingredients in the study medication formulation. 4. The participant is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests. 5. The participant has uncontrolled systemic disease. 6. The participant has been treated for invasive cancer within the past 5 years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) Stage 0. 7. The participant 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 participation in an experimental drug study while enrolled in this study. 8. The participant is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures. 9. The participant is pregnant or breastfeeding. 10. The participant has any condition or situation which, in the Investigator's opinion, may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study. This may include a history of other skin conditions (such as severe facial eczema) or diseases, metabolic dysfunction, physical examination findings, or clinical laboratory findings giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the participant at high risk from treatment complications.

Contacts and Locations

Sponsors and CollaboratorsSol-Gel Technologies, Ltd.
Locations
Mayo Clinical Cancer Center | Phoenix Arizona, United States, 85054Dermatology Center of Newport | Newport Beach California, United States, 92660Stanford University, Department of Dermatology | Redwood City California, United States, 94063University of California, San Francisco | San Francisco California, United States, 94115Yale School of Medicine | New Haven Connecticut, United States, 06519University of Miami Miller School of Medicine | Miami Florida, United States, 33136Leavitt Medical Associates of Florida | Ormond Beach Florida, United States, 32174The University of Chicago | Chicago Illinois, United States, 60637Laser & Skin Surgery Center of Indiana | Carmel Indiana, United States, 46032Dana-Farber Cancer Institute | Boston Massachusetts, United States, 02215University of Michigan, Dept of Dermatology | Ann Arbor Michigan, United States, 48109-5314University of Minnesota, Department of Dermatology | Minneapolis Minnesota, United States, 55455Saint Louis University Dermatology | St Louis Missouri, United States, 63104Dartmouth-Hitchcock Medical Center | Lebanon New Hampshire, United States, 03756Columbia University Medical Center | New York New York, United States, 10032Duke University | Durham North Carolina, United States, 27710Cleveland Clinic Foundation | Cleveland Ohio, United States, 44195Oregon Health and Science University | Portland Oregon, United States, 97239Penn State Health Milton S. Hershey Medical Center | Hershey Pennsylvania, United States, 17033Hospital of the University of Pennsylvania | Philadelphia Pennsylvania, United States, 19104MD Anderson Cancer Center | Houston Texas, United States, 77030University of Utah Midvalley Dermatology | Murray Utah, United States, 84107PellePharm Investigative Site | Brussels , Belgium, 1200PellePharm Investigative Site | Leuven , Belgium, 3000Clinical Trials Unit - The Skin Care Centre | Vancouver British Columbia, Canada, Pellepharm Investigative Site | Toronto , Canada, M5S1B2PellePharm Investigative Site | Copenhagen , Denmark, Hopital Saint-Andre - CHU Bordeaux | Bordeaux , France, 33075CHRU de Lille - Hopital Claude HURIEZ | Lille , France, 59037CHU La Timone | Marseille , France, 13395PellePharm Investigative Site | Nantes , France, 44093Hopital Saint-Louis | Paris , France, 75010PellePharm Investigative Site | Pierre-Bénite , France, 69310Charite - Universitatsmedizin Berlin | Berlin , Germany, 10117Klinik und Poliklinik fur Dermatologie und Allergologie | Munich , Germany, 80337Universitatsklinikum Münster | Münster , Germany, 48149Universitats Hautklinik | Tübingen , Germany, 72076Ospedale Vanvitelli-University della Campania-Dermatologia Edificio 9 | Naples , Italy, 80131Catholic University of the Sacred Heart | Roma , Italy, 00168Humanitas University Milan | Rozzano , Italy, 20089Ospedale San Bortolo | Vicenza , Italy, 36100Maastricht University Medical Center - Dept of Dermatology | Maastricht , Netherlands, 6202Hospital Clinic I Provincial | Barcelona , Spain, 08036Hospital Universitario Ramon y Cajal | Madrid , Spain, 28034Hospital Universitiario Virgen de la Macarena | Seville , Spain, 41009NHS Greater Glasgow and Clyde | Glasgow , United Kingdom, G38SJRoyal London Hospital | London , United Kingdom, E1 1BBOxford University Hospitals NHS Foundation Trust | Oxford , United Kingdom, OX37LESalford Royal Hospital | Salford , United Kingdom, M68HD
Investigators
Study Director: VP, Clinical Operations, PellePharm, Inc.
Study Documents (Full Text)
Documents provided by Sol-Gel Technologies, Ltd.: Study Protocol  January 3, 2021Documents provided by Sol-Gel Technologies, Ltd.: Statistical Analysis Plan  June 16, 2020