E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Fungal infection of the nails |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10030338 |
E.1.2 | Term | Onychomycosis |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
|
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 evaluate the tolerability and safety of topical treatment with P-3058 10% o.w. in pediatric patients with onychomycosis. |
|
E.2.2 | Secondary objectives of the trial |
- Overall safety evaluation by recording all adverse event
occurring during the study; - Acceptance of therapy by children/Parents or Guardians at the end of treatment; - Complete cure defined as composite of negative KOH microscopy and negative culture for dermatophytes and no residual clinical involvement of the target nail at all study visits; - Responder rate defined as negative KOH microscopy and
negative culture for dermatophytes and ≤10% residual
involvement of the target nail at all study visits; - Conversion to negative KOH microscopy at all study visits; - Conversion to negative culture for dermatophytes at all study visits; - Conversion to negative mycology (combination of negative KOH microscopy and negative dermatophytes culture); - Rate of patients having diseased nail ≤10% or 0% (Clinical
Cure); - Decrease of the target nail area at all study visits; - Time to reach the efficacy outcome cure/responder rate. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Written Informed Consent given by the patient and
parents/Guardians and in any case before starting any study
related procedures; 2. Patients of both genders, aged between 2 and 17 years at the time of the written informed consent with established clinical diagnosis of mild-to-moderate distal sub-ungual onychomycosis (DSO) without spikes/dermatophytoma and without lunula involvement or white superficial onychomycosis (WSO); 3. Patients with positive KOH examination from the target nail chosen at screening; 4. Patients with positive culture for dermatophyte from the target nail chosen at screening. |
|
E.4 | Principal exclusion criteria |
1. Patients with history of allergic reactions to terbinafine and/or to
any of the other ingredients of P-3058; 2. Patients with history of cardiovascular, renal, neurologic, liver, immunologic or endocrine dysfunction if they are clinically significant; 3. Patients with onychomycosis caused by yeasts or nondermatophytes mould; 4. Patients with severe plantar or moccasin tinea pedis (defined by blistering, pustules or inability to ambulate). Patients with mildto-moderate tinea pedis using a topical antifungal treatment will be included; 5. Congenital or acquired nail dystrophy (i.e. Darier’s disease); 6. Patients with nail psoriasis; 7. Patients with nail changes due to eczema, lichen planus or alopecia areata; 8. Patients with one-hand two-foot syndrome; 9. Patients with immunodeficiency disorder or use of immune suppressive therapy 3 months prior to screening visit or need for it; 10. Use of systemic antifungal drugs in the 6 months prior to screening visit; 11. Use of topical nail antifungal drugs in the four weeks prior to screening visit; 12. Habitual finger sucking during the night in children that have fingernail onychomycosis if it cannot be prevented by parents; 13. Female subjects physiologically capable of becoming pregnant are asked to refrain from any sexual intercourse during the whole study duration or to use acceptable methods of contraception
agreed with the investigator; 14. Female subjects who are pregnant, nursing mothers, those planning a pregnancy during the course of the study or who
become pregnant. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Local tolerability at the application site (all treated nails) during the
study period by means of the Severity Score for Skin Irritation. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- Overall safety evaluation by recording all adverse event
occurring during the study; - Acceptance of therapy by children/Parents or Guardians at the end of treatment; - Complete cure defined as composite of negative KOH microscopy and negative culture for dermatophytes and no residual clinical involvement of the target nail at all study visits; - Responder rate defined as negative KOH microscopy and
negative culture for dermatophytes and ≤10% residual
involvement of the target nail at all study visits; - Conversion to negative KOH microscopy at all study visits; - Conversion to negative culture for dermatophytes at all study visits; - Conversion to negative mycology (combination of negative KOH microscopy and negative dermatophytes culture); - Rate of patients having diseased nail ≤10% or 0% (Clinical
Cure); - Decrease of the target nail area at all study visits; - Time to reach the efficacy outcome cure/responder rate. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- during the study; - at the end of treatment; - at all study visits; - at all study visits; - at all study visits; - at all study visits; - during the study; - during the study; - at all study visits; - during the study. |
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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 | Yes |
E.6.13.1 | Other scope of the trial description |
|
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
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
|
The end of study is defined as the date of database locked. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |