E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Basal cell carcinomas (BCCs) in patients with Gorlin syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Skin cancer in patients with Gorlin syndrome |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10004151 |
E.1.2 | Term | Basal cell nevus syndrome |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of the study is to assess the safety and tolerability of Patidegib Topical Gel, 2% in patients who have completed PellePharm Study Pelle-926-201 or Pelle-926-301 |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of the study is to assess the efficacy of Patidegib Topical Gel, 2% in patients who complete PellePharm Study Pelle-926-201 or Pelle-926-301 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The subject must be at least 18 years old. 2. The subject must provide written informed consent prior to any study procedures. 3. The subject must have completed PellePharm Study 926-201 or 926-301 with adequate compliance 4. Study 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. 5. The subject must meet diagnostic criteria for Gorlin (basal cell nevus) syndrome, including major criterion #a plus 1 additional major criterion or plus 2 additional minor criteria listed in Appendix 17.2 6. 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. 7. Female subjects must have a negative pregnancy test (serum pregnancy test at Screening Visit, urine pregnancy test at Baseline Visit). 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. 8. 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 (Appendix 17.1). 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 IP 9. 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. 10. The subject is willing for all facial BCCs to be evaluated and follow treatment recommendations made only by the Investigator 11. 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. |
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E.4 | Principal exclusion criteria |
1. The subject 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: a. 5-fluorouracil, imiquimod, or Ingenol mebutate (except as topical treatment to anatomical areas other than the face) systemically or topically to the skin within the 2 months prior to the Screening and Baseline Visit. b. Systemic chemotherapy within 1 year prior to the Screening and Baseline Visit. c. Known inhibitors of the Hedgehog signaling pathway (e.g., vismodegib, sonidegib, itraconazole) topically (except as topical treatment to anatomical areas other than the face) or systemically within 2 months prior to the Screening and Baseline Visit. d. Photodynamic therapy (PDT) except to localized non-facial, individual BCCs within 2 months prior to the Screening and Baseline Visit. 2. The subject has a known hypersensitivity to any of the ingredients in the study IP formulation. 3. The subject is unable or unwilling to make a good faith effort to return to the study site for all study visits and tests. 4. 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 participation in an experimental drug study (excluding Study 926-301) while enrolled in this study. 5. The subject is a WOCBP who is unwilling or unable to comply with pregnancy prevention measures. 6. The subject is pregnant or breastfeeding. 7. The subject 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 (e.g., severe facial eczema) or diseases, metabolic dysfunction, physical examination (PE) 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 subject at high risk from treatment complications. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The number of facial BCCs removed by surgery is considered an appropriate study endpoint as surgery has significant morbidity in Gorlin Syndrome (e.g., scarring, functional loss of eyelid, nose, ear). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
The efficacy endpoints are as follows: 1. The number of facial BCCs removed by surgery (per Investigator determination) and histologically verified as BCC; from Baseline to Month 12 2. The number of facial BCCs removed by surgery (per Investigator determination) and histologically verified as BCC; from Baseline to Month 6. 3. The number of facial BCCs removed by surgery (per Investigator determination) from Baseline to Month 12. 4. The number of facial BCCs removed by surgery (per Investigator determination) from Baseline to Month 6. 5. Change from Baseline to Month 12 in the total number of facial lesions suspicious for BCC 6. Count of facial lesions suspicious for BCC over time 7. aBCCdex change in lesion score from Baseline to Month 12 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 22 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Canada |
Denmark |
France |
Germany |
Italy |
Netherlands |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 13 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 16 |