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

NCT04308395 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: Pelle-926-301E
• Study Type: interventional
• Target: 108
• Locations: 9 countries
• Sites: 37

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.

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

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (5)

• 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

Study Arms (1)

Patidegib Topical Gel, 2%

EXPERIMENTAL

Patidegib Topical Gel, 2%

Drug: Patidegib Topical Gel, 2%

Interventions

Patidegib Topical Gel, 2% DRUG

Patidegib Topical Gel, 2%

Patidegib Topical Gel, 2%

Eligibility Criteria

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

You may qualify if:

• The subject must have completed PellePharm Study Pelle-926-201 or Pelle-926-301.
• 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.
• 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.
• 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.
• 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).
• 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.
• The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator.
• 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.

You may not qualify if:

• The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study IP.
• 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).
• The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures.
• The subject is pregnant or breastfeeding.

Sponsors & Collaborators

• Sol-Gel Technologies, Ltd.: lead

Study Sites (37)

PellePharm Investigative Site

Fremont, California, 94538, United States

Location

PellePharm Investigative Site

Newport Beach, California, 92660, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

Pellepharm Investigative Site

Miami, Florida, 33125, United States

Location

PellePharm Investigative Site

Ormond Beach, Florida, 32174, United States

Location

PellePharm Investigative Site

Chicago, Illinois, 60637, United States

Location

Laser & Skin Surgery Center of Indiana

Indianapolis, Indiana, 46260, United States

Location

Pellepharm Investigative Site

Ann Arbor, Michigan, 48109, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Saint Louis University

St Louis, Missouri, 63104, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

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

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Utah, Midvalley Dermatology

Murray, Utah, 84107, United States

Location

PellePhram Investigative Site

Leuven, 3000, Belgium

Location

PellePharm Investigative Site

Copenhagen, DK-2400, Denmark

Location

PellePharm Investigative Site

Lille, France

Location

PellePharm Investigative Site

Nantes, 44093, France

Location

Hopital Saint Louis

Paris, 75010, France

Location

PellePharm Investigative Site

Berlin, 10117, Germany

Location

PellePharm Investigative Site

Munich, 80337, Germany

Location

PellePharm Investigative Site

Münster, 48149, Germany

Location

AOU Luigi Vanvitelli

Napoli, 80131, Italy

Location

PellePharm Investigative Site

Rozzano, 20089, Italy

Location

PellePharm Investigative Site

Vicenza, 36100, Italy

Location

PellePharm Investigative Site

Maastricht, 6229 HX, Netherlands

Location

PellePharm Investigative Site

Barcelona, 08036, Spain

Location

PellePharm Investigative Site

Madrid, 28023, Spain

Location

PellePharm Investigative Site

Madrid, 28034, Spain

Location

PellePharm Investigative Site

Seville, 41009, Spain

Location

PellePharm Investigative Site

Glasgow, United Kingdom

Location

PellePharm Investigative Site

London, United Kingdom

Location

PellePharm Investigative Site

Manchester, United Kingdom

Location

PellePharm Investigative Site

Oxford, 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

• Study Director 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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All patients will receive Patidegib Topical Gel, 2%,

Study Record Dates

• First Submitted: March 11, 2020
• First Posted: March 16, 2020
• Study Start: June 3, 2020
• Primary Completion: July 14, 2021
• Study Completion: July 14, 2021
• Last Updated: September 24, 2024
• Results First Posted: September 24, 2024

Record last verified: 2024-07

Locations

FR (3); ES (4); US (17); NL (1); DE (3); DK (1); GB (4); IT (3); BE (1)