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)

NCT03703310 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: Pelle-926-301
• Study Type: interventional
• Target: 174
• Locations: 10 countries
• Sites: 49

Brief Summary

This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period.

Conditions

Basal Cell Nevus Syndrome

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number of New BCCs Per Participant

  Month 12

Secondary Outcomes (6)

• Number of New Surgically Eligible BCCs (nSEBs) Per Participant

  Month 12

• Percentage of Participants Developing >=2 Facial New BCCs

  Month 12

• Percentage of Participants Developing >=1 Facial New BCCs

  Month 12

• Number of New BCCs Per Participant

  Month 9

• Number of New BCCs Per Participant

  Month 6

Study Arms (2)

Patidegib Topical Gel, 2%,

EXPERIMENTAL

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, Vehicle

PLACEBO COMPARATOR

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

Interventions

Patidegib Topical Gel, 2% DRUG

Patidegib Topical Gel, 2%

Also known as: IP

Patidegib Topical Gel, 2%,

Patidegib Topical Gel, Vehicle DRUG

Patidegib Topical Gel, Vehicle

Also known as: IP, Vehicle

Patidegib Topical Gel, Vehicle

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The participant must be age at least 18 years of age at the Screening Visit.
• The participant must provide written informed consent prior to any study procedures.
• 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:
• \>2 histologically confirmed BCCs or 1 for participant under age 20.
• Odontogenic keratocysts of the jaw confirmed histologically.
• ≥3 palmar and/or plantar pits seen at the Screening Visit.
• Bilamellar calcification of the falx cerebri present at less than 20 years of age.
• Fused, bifid, or markedly splayed ribs.
• First degree relative with Gorlin syndrome.
• Patched protein 1 (PTCH1) mutation predicted to be of functional significance in normal tissue.
• Minor criteria:
• Macrocephaly.
• Congenital malformations including frontal bossing, cleft lip or palate, "coarse face", moderate to severe hypertelorism.
• Skeletal abnormalities detectable clinically: Sprengel deformity, marked pectus deformity, or marked finger syndactyly.

You may not qualify if:

• The subject has previously participated in a clinical trial evaluating patidegib topical gel.
• 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:
• 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.
• Systemic chemotherapy within 1 year prior to the Screening Visit.
• Known inhibitors of the Hedgehog signaling pathway (such as vismodegib, sonidegib, itraconazole) topically or systemically within 3 months prior to the Screening Visit.
• Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 2 months prior to the Screening Visit.
• The participant is known to have a hypersensitivity to any of the ingredients in the study medication formulation.
• The participant is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests.
• The participant has uncontrolled systemic disease.
• 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.
• 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.
• The participant is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
• The participant is pregnant or breastfeeding.
• 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.

Sponsors & Collaborators

• Sol-Gel Technologies, Ltd.: lead

Study Sites (49)

Mayo Clinical Cancer Center

Phoenix, Arizona, 85054, United States

Location

Dermatology Center of Newport

Newport Beach, California, 92660, United States

Location

Stanford University, Department of Dermatology

Redwood City, California, 94063, United States

Location

University of California, San Francisco

San Francisco, California, 94115, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Leavitt Medical Associates of Florida

Ormond Beach, Florida, 32174, United States

Location

The University of Chicago

Chicago, Illinois, 60637, United States

Location

Laser & Skin Surgery Center of Indiana

Carmel, Indiana, 46032, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan, Dept of Dermatology

Ann Arbor, Michigan, 48109-5314, United States

Location

University of Minnesota, Department of Dermatology

Minneapolis, Minnesota, 55455, United States

Location

Saint Louis University Dermatology

St Louis, Missouri, 63104, United States

Location

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Utah Midvalley Dermatology

Murray, Utah, 84107, United States

Location

PellePharm Investigative Site

Brussels, 1200, Belgium

Location

PellePharm Investigative Site

Leuven, 3000, Belgium

Location

Clinical Trials Unit - The Skin Care Centre

Vancouver, British Columbia, Canada

Location

Pellepharm Investigative Site

Toronto, M5S1B2, Canada

Location

PellePharm Investigative Site

Copenhagen, Denmark

Location

Hopital Saint-Andre - CHU Bordeaux

Bordeaux, 33075, France

Location

CHRU de Lille - Hopital Claude HURIEZ

Lille, 59037, France

Location

CHU La Timone

Marseille, 13395, France

Location

PellePharm Investigative Site

Nantes, 44093, France

Location

Hopital Saint-Louis

Paris, 75010, France

Location

PellePharm Investigative Site

Pierre-Bénite, 69310, France

Location

Charite - Universitatsmedizin Berlin

Berlin, 10117, Germany

Location

Klinik und Poliklinik fur Dermatologie und Allergologie

Munich, 80337, Germany

Location

Universitatsklinikum Münster

Münster, 48149, Germany

Location

Universitats Hautklinik

Tübingen, 72076, Germany

Location

Ospedale Vanvitelli-University della Campania-Dermatologia Edificio 9

Napoli, 80131, Italy

Location

Catholic University of the Sacred Heart

Roma, 00168, Italy

Location

Humanitas University Milan

Rozzano, 20089, Italy

Location

Ospedale San Bortolo

Vicenza, 36100, Italy

Location

Maastricht University Medical Center - Dept of Dermatology

Maastricht, 6202, Netherlands

Location

Hospital Clinic I Provincial

Barcelona, 08036, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitiario Virgen de la Macarena

Seville, 41009, Spain

Location

NHS Greater Glasgow and Clyde

Glasgow, G38SJ, United Kingdom

Location

Royal London Hospital

London, E1 1BB, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, OX37LE, United Kingdom

Location

Salford Royal Hospital

Salford, M68HD, United Kingdom

Location

MeSH Terms

Conditions

Basal Cell Nevus Syndrome; Carcinoma, Basal Cell

Condition Hierarchy (Ancestors)

Odontogenic Cysts; Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Jaw Diseases; Stomatognathic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Results Point of Contact

• Title: VP Clinical & Regulatory Affairs
• Organization: Sol-Gel Technologies, Ltd.

Ofra.Levy-Hacham@sol-gel.com

+972-73-3729453

Study Officials

• VP, Clinical Operations

  PellePharm, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: As a double-blinded study, the Investigators, the site staff, Sponsor, and the Clinical Monitor(s) will be blinded to the treatment assigned to individual participants.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be randomized 1:1 to receive Patidegib Topical Gel, 2%, or Vehicle (IP)

Study Record Dates

• First Submitted: October 9, 2018
• First Posted: October 11, 2018
• Study Start: February 19, 2019
• Primary Completion: December 28, 2020
• Study Completion: December 28, 2020
• Last Updated: July 3, 2024
• Results First Posted: July 3, 2024

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (4); GB (4); BE (2); DK (1); ES (3); CA (2); IT (4); US (22); FR (6); NL (1)