E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002461 |
E.1.2 | Term | Angioma of skin |
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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 compare the efficacy one month after the end of the treatment, between the association of timolol gel and pulsed dye laser (PDL), and PDL alone in the treatment of port wine stains in children above 6 months. |
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E.2.2 | Secondary objectives of the trial |
To study the satisfaction of the parents of the children (and if possible also of the patients themselves depending on their age) on the efficacy and the tolerance of the treatments. To study the possible occurrence of side effects.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Children from 6 months to 18 year-old with a port wine stain of the face - Signed agreement of the parents, and of the child if he is old enough to understand - Affiliation to the social health care program.
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E.4 | Principal exclusion criteria |
- Port wine stain already treated with a laser or an intense pulsed light - Personal history of asthma or obstructive bronchitis - Severe allergic rhinitis - Sinusal bradycardia, second and third degree atrioventricular block - Cardiac decompensation - Untreated pheochromocytoma - Hypersensitivity to timolol or to one of its component, and/or to other beta-blocker - Concomitant treatment with floctafenine or with sultopride - Oral treatment with beta-blocker, or calcium-channel blocker, or amiodarone
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E.5 End points |
E.5.1 | Primary end point(s) |
The response or therapeutic success will be defined clinically as a marked improvement or a complete regression thanks to an investigator global assessment (IGA) score ≥ 3 (cf. annex 1), one month after the last PDL session. The area treated will be located thanks to anatomical marks. Standardized pictures will be done before and at the end of the study. The evaluation will be blinded and performed by two independent physicians. In case of disagreement a third observer will be required. |
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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 | No |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
Laser + Neogel vs Laser also |
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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 | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | 5 |
E.8.9.1 | In the Member State concerned days | |