E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Basal Cell Nevus Syndrome |
Sindrome del nevo basocellulare |
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E.1.1.1 | Medical condition in easily understood language |
Gorlin Syndrom |
Sindrome di Gorlin |
|
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 |
|
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 |
|
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. |
Valutare il numero di nuovi carcinomi basocellulari (Basal Cell Carcinoma, BCC) operabili (New Surgically Eligible BCC, nSEB) nei 2 bracci (patidegib gel topico, 2% e veicolo) con applicazione due volte al giorno sul viso di soggetti con sindrome del nevo basocellulare (Basal Cell Nevus Syndrome, BCNS). |
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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. |
Valutare la sicurezza e la tollerabilità di patidegib gel topico, 2% in soggetti trattati due volte al giorno per 12 mesi. |
|
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. Il soggetto deve avere almeno 18 anni alla visita di screening. 2. Il soggetto deve soddisfare i criteri diagnostici della BCNS (criterio di inclusione 3). 3. Il soggetto deve aver avuto almeno 10 BCC (di cui almeno 3 localizzati sul viso) con presentazione clinica tipica nei 24 mesi precedenti la randomizzazione (basale/Giorno 1). Il soggetto deve inoltre avere almeno 2 BCC di diametro massimo < 5 mm sul viso prima della randomizzazione. 4. Il soggetto deve essere disposto ad astenersi dall’applicazione di medicinali topici (su prescrizione o da banco) diversi da quelli previsti dallo studio sulla cute del viso per l’intera durata dello studio.
L’elenco completo dei criteri di inclusione ed esclusione è riportato nella sezione 7.1. |
|
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. Il soggetto ha fatto uso di un trattamento topico sul viso o di terapie sistemiche che potrebbero interferire con la valutazione dell’IP dello studio. 2. Il soggetto presenta un’ipersensibilità nota a uno qualsiasi dei componenti dell’IP. 3. Il soggetto presenta una patologia sistemica non controllata. 4. Il soggetto ha ricevuto una terapia per cancro invasivo nei 5 anni precedenti, esclusi carcinoma cutaneo non melanoma, cancro della cervice uterina in stadio I, carcinoma duttale in situ della mammella o leucemia linfocitica cronica (LLC) in stadio 0.
L’elenco completo dei criteri di inclusione ed esclusione è riportato nella sezione 7.1. |
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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. |
L'endpoint primario è il numero di nSEB a 12 mesi. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
1. The number of new BCCs that develop from Baseline to Month 12 2. The proportion of subjects developing =2 facial nSEB(s) by Month 12. 3. The proportion of subjects developing =1 facial nSEB(s) by Month 12. 4. The number of qualifying nSEBs per subject at Month 9. 5. The number of qualifying nSEBs per subject at Month 6. 6. aBCCdex change in lesion score from Baseline to Month 12. |
1. Il numero di nuovi BCC che si sviluppano dal basale al mese 12 2. La proporzione di soggetti che sviluppano =2 nSEB facciali entro il mese 12. 3. La proporzione di soggetti che sviluppano =1 nSEB facciale entro il mese 12. 4. Il numero di nSEB qualificati per soggetto al mese 9. 5. Il numero di nSEB qualificati per soggetto al mese 6. 6. aBCCdex cambia il punteggio della lesione dal basale al mese 12. |
|
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. 12 mesi 2. 12 mesi 3. 9 mesi 4. 6 mesi 5. 12 mesi 6. 12 mesi |
|
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 | 6 |
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 | Information not present in EudraCT |
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 |
Canada |
United States |
Belgium |
Denmark |
France |
Germany |
Italy |
Netherlands |
Spain |
United Kingdom |
|
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
|
|
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 |