Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Ce… (NCT02762084) | Clinical Trial Compass
CompletedPhase 2
Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
United Kingdom17 participantsStarted 2016-06-06
Plain-language summary
Multicenter, double-blind, randomized, vehicle-controlled study that evaluates the efficacy and safety of patidegib gel 2% and 4% in comparison with vehicle in participants at least 18 years of age that meet the diagnostic criteria for basal cell nevus syndrome (BCNS). Participants will be randomized to receive patidegib gel 2%, patidegib gel 4%, or the vehicle gel for a 26-week treatment period.
Who can participate
Age range18 Years – 85 Years
SexALL
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Inclusion criteria
✓. The participant is from 18 to 85 years of age, inclusive.
✓. The participant must provide written informed consent prior to any study procedures.
✓. The participant must meet diagnostic criteria for BCNS, including the first listed major criterion below plus one additional major criterion, or the first listed major criterion below plus 2 of the minor criteria outlined below:
✓. The participant must have a history of at least 10 BCCs in toto present at Baseline and/or treated within 24 months prior to screening.
✓. The participant has at Baseline a total of at least 5 previously untreated SEBs (greatest diameter 5 millimeters \[mm\] or greater on the face excluding the nose and periorbital skin, 9 mm or greater on non-facial areas excluding the skin below the knees), as documented clinically by the Investigator at Baseline. Untreated is define as no previous surgical or topical or intralesional drug treatment. Previous treatment with systemically administered drugs more than 6 months prior to Baseline is not considered previous treatment as long as there was no clinical evidence of resistance to oral hedgehog (HH) pathway inhibitors (such as vismodegib, patidegib, and sonidegib). Baseline treatment-targeted SEBs must not exceed a diameter of \>2 centimeters (cm). At least one of these tumors must be appropriate for a 2 mm punch biopsy for biomarker analysis at Baseline and Week 6 visits. If a participant has 5 or more facial, excluding periorbital and nasal skin, SEBs at Baseline, non-facial SEBs will not be treatment-targeted SEBs.
✓. The participant is willing to have SEBs biopsied for biomarkers and plasma to be collected to measure drug levels as required in the protocol.
✓. The participant is willing to abstain from application of non-study topical prescription and over the counter medications to facial skin and within 5 cm of treatment targeted SEBs at other anatomical areas for the duration of the study except as prescribed by the Investigator. Moisturizers and emollients are allowable. Participants will be encouraged to use sunscreen with a sunscreen protection factor (SPF) 15 or higher at least once daily on all exposed skin sites.
What they're measuring
1
Clinical Efficacy: Percent Change in Tumor Size of Treatment-targeted Surgically Eligible Basal Cell Carcinomas (SEBs) From Baseline
Timeframe: Baseline, Week 26
2
Molecular Efficacy: Percent Change in the Hedgehog (HH) Signaling Pathway Target Gene Glioma-associated Oncogene Homolog 1 (GLI1) Messenger Ribonucleic Acid (mRNA) Levels From Baseline
Timeframe: Baseline, Week 6
3
Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With a Treatment-emergent Adverse Event Causally Related to Study Drug
Timeframe: Baseline through Week 26
4
Safety and Tolerability Assessment of Treatment With Patidegib Gel: Number of Participants With Treatment-emergent Administrative Site Skin Condition AEs Causally Related to Study Drug
✓. Female participants must have a negative serum pregnancy test at Screening.
Exclusion criteria
✕. The participant is a woman of childbearing potential. This proscription is based on the key role of the HH pathway in embryogenesis, the known preclinical teratogenic effects of systemic cyclopamine, a naturally occurring inhibitor of SMO, and the unknown level of systemic exposure following topical application in humans. A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (that is, has had menses at any time in the preceding 24 consecutive months).
✕. The participant has used topical products to the face or within 5 cm of a treatment targeted SEB or systemic therapies that might interfere with the evaluation of the study medication during the study. Specifically, these include the use of:
✕. The participant has a history of hypersensitivity to any of the ingredients in the study drug formulation.
✕. The participant is unable or unwilling to make a good faith effort to return for all follow-up visits and tests.
✕. The participant has uncontrolled systemic disease.
✕. The participant has clinically important history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis.
✕. The participant has any condition or situation which in the Investigator's opinion may put the participant at significant risk, could confound the study results, or could interfere significantly with the participant's participation in the study. This includes history of other skin conditions 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.
✕. The participant has a history of invasive cancer within the past 5 years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of breast, or chronic lymphocytic lymphoma (Stage 0).