E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Basal Cell Nevus Syndrome |
Síndrome basocelular nevoide |
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E.1.1.1 | Medical condition in easily understood language |
Gorlin Syndrom |
Síndrome de Gorlin |
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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 number of new surgically eligible BCCs (nSEBs) in the 2 arms (Patidegib Topical Gel, 2%, and Vehicle) when applied twice daily to the face of subjects with BCNS |
El objetivo principal del estudio es determinar el número de nuevos CBC aptos para cirugía (nAPTOSc) en los dos grupos (patidegib gel tópico al 2 % y vehículo) tras su aplicación dos veces al día en la cara de sujetos con SNBC |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study is to assess the safety and tolerability of Patidegib Topical Gel, 2%, in subjects treated twice daily for 12 months |
El objetivo secundario es evaluar la seguridad y la tolerabilidad de patidegib gel tópico al 2 % en sujetos tratados dos veces al día durante 12 meses. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The subject must be at least 18 years old at the Screening visit. 2. The subject must meet diagnostic criteria for BCNS (Inclusion Criteria 3). 3. The subject must have had at least 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. 4. 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. |
1.Los sujetos deben tener como mínimo 18 años de edad en el momento de la visita de selección. 2.Los sujetos deben cumplir los criterios de diagnóstico de SNBC (criterio de inclusión 3). 3.Los sujetos deben tener al menos diez (y al menos tres en la cara) CBC clínicamente típicos durante los 24 meses previos a la aleatorización (visita inicial/día 1). Además, los sujetos deben tener al menos dos CBC con el diámetro mayor < 5 mm en la cara antes del momento de la aleatorización. 4.Los sujetos deben estar dispuestos a abstenerse de aplicarse medicamentos tópicos que no sean los del estudio (ya sean recetados o de venta sin receta) en la piel de la cara durante todo el estudio. |
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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. 2. The subject is known to have a hypersensitivity to any of the ingredients in the IP. 3. The subject has uncontrolled systemic disease. 4. The subject 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. |
1.El sujeto se ha aplicado un tratamiento tópico en la cara o ha tomado tratamientos sistémicos que podrían interferir con la evaluación del PEI del estudio. 2.Se sabe que el sujeto presenta hipersensibilidad a cualquiera de los componentes del PEI. 3.El sujeto presenta una enfermedad sistémica no controlada. 4.El sujeto ha recibido tratamiento para un cáncer invasivo en los cinco últimos años, con la excepción de cáncer de piel no melanocítico, cáncer cervicouterino en estadio I, carcinoma ductal in situ de la mama o leucemia linfocítica crónica (LLC) en estadio 0. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the number of nSEBs at 12 months |
El objetivo principal es el número de nAPTOSc a los 12 meses |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Month 12 (Week 52) |
Mes 12 (semana 52) |
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E.5.2 | Secondary end point(s) |
Secondary Endpoints: 1. The proportion of subjects developing ≥2 facial nSEB(s) by Month 12. 2. The proportion of subjects developing ≥1 facial nSEB(s) by Month 12. 3. The number of qualifying nSEBs per subject at Month 9. 4. The number of qualifying nSEBs per subject at Month 6. 5.The number of new BCCs that develop from Baseline to Month 12. 6.aBCCdes change in lesion score from Baseline to Month 12. |
Criterios de valoracón secundarios: 1. La proporción de sujetos que desarrollan ≥2 nAPTOSc faciales hasta el mes 12. 2. La proporción de sujetos que desarrollan ≥1 nAPTOSc faciales hasta el mes 12. 3. El número de nAPTOSc cualificados por sujeto al mes 9. 4. El número de nAPTOSc cualificados por sujeto al mes 6. 5. El número de nuevos CBCs que se desarrollan desde el inicio hasta el mes 12. 6. Cambio en la puntuación de la lesion de in CBCs desde el inicio hasta el mes 12. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Month 12 2. Month 12 3. Month 9 4. Month 6 5. Month 12 6. Month 12 |
1. Mes 12 2. Mes 12 3. Mes 9 4. Mes 6 5. Mes 12 6. Mes 12 |
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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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 30 |
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 |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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
|
LPLV |
última visista del ultimo paciente |
|
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 | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |