E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients over 18 years of age with a previously untreated superficial or nodular basal cell carcinoma (BCC) on the scalp, face, extremities or trunk |
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E.1.1.1 | Medical condition in easily understood language |
Patients over 18 years of age with a previously untreated skin cancer lesion (basal cell carcinoma) on the scalp, face, extremities or trunk |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10004146 |
E.1.2 | Term | Basal cell carcinoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To investigate tolerability (i.e. local skin reactions and side effects) of ablative fractional laser-assisted cisplatin+5-FU therapy for basal cell carcinoma |
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E.2.2 | Secondary objectives of the trial |
To investigate change in tumor size and clearance following ablative fractional laser-assisted cisplatin+5-FU therapy for basal cell carcinoma |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects who meet all of the following criteria are eligible to participate in this study: 1. Histologically-verified, previously untreated superficial or nodular BCCs on the scalp, face, extremities or trunk 2. >18 years of age at baseline 3. Legally competent, able to give verbal and written consent 4. Subject in good general health, is willing to participate and able to give informed consent, and can comply with protocol requirements. 5. Fitzpatrick skin phototype I-III 6. Female subjects of childbearing potential must be confirmed not pregnant by a negative urine pregnancy test prior to trial treatment.
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E.4 | Principal exclusion criteria |
Subjects meeting any of the following criteria are not eligible to participate in this study:
1. High-risk BCC: i. Tumors in the following anatomical locations: midface, orbital, ears ii. Size: >20 mm in facial/scalp areas or > 50mm in non-facial/non-scalp areas iii. Subtype: Mopheraform or micronodular iv. History: Gorlin syndrome or immunesuppression
2. Previous treatment of the BCC lesion 3. Known allergy to cisplatin or Efudix® 4. Other skin diseases in the area of interest 5. Tattoo in the treatment area which may interfere with or confound evaluation of the study 6. History of keloids which is deemed clinically relevant in the opinion of the investigator 7. Fitzpatrick skin phototype IV-VI 8. Lactating or pregnant women |
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E.5 End points |
E.5.1 | Primary end point(s) |
Tolerability measured as non-blinded, clinical evaluation of:
I) Local skin reactions (LSRs): erythema, edema, flaking, crusting/scabbing and pustulation
II) Local side effects: prolonged erythema or edema, hyper- or hypopigmentation, scarring and infection |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Local skin reactions (LSRs): Days 1, 5 and 14 after baseline treatment
Side effects: Up to 3 months after baseline treatment |
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E.5.2 | Secondary end point(s) |
Tumor Response measured as:
I) Tumor size (mm) and clearance (yes or no) will be evaluated clinically and using non-invasive dynamic optical coherence tomography (OCT), high intensity focused ultrasound (HIFU) and reflectance confocal microscopy (RCM) imaging at baseline, Day 30 (not RCM) and Month 3.
II) Histological verification of tumor clearance will be performed 3 months after first treatment using tissue sections from a 4 mm punch biopsy. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
I) Tumor size (mm) and clearance (yes or no) will be evaluated at baseline, Day 30 and Month 3
II) Histological verification of tumor clearance will be performed 3 months after baseline treatment |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
All patients receieve treatment with ablative fractonal laser followed by topical cisplatin and 5-FU |
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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.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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 | |
E.8.9.1 | In the Member State concerned days | |