E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polymorphic light eruption (PLE) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036087 |
E.1.2 | Term | Polymorphic light eruption |
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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 |
Primary objective To determine whether afamelanotide can reduce the severity of outbreaks of PLE. |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives To determine whether afamelanotide can reduce the number of outbreaks of PLE. To evaluate the safety and tolerability of afamelanotide by measuring treatment-emergent adverse events. To evaluate the effect of afamelanotide on the use of rescue medication for outbreaks of PLE To evaluate the effect of afamelanotide on the quality of life of PLE patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To be eligible to enter the study, patients must meet the following inclusion criteria: - Aged greater than 18 years - Male or female patients with a documented history of moderate/severe PLE diagnosed or confirmed by a photodermatologist or photobiologist. - Recurrent PLE episodes that occur at least once a year, developing in own country (to exclude patients affected only when traveling to sunnier climates). - Written informed consent prior to the performance of any study-specific procedure - Are willing and able to comply with the conditions specified in this protocol and study procedures in the opinion of the Investigator.
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E.4 | Principal exclusion criteria |
To be eligible to enter the study, patients must not meet any of the following exclusion criteria: - Personal history of melanoma, lentigo maligna or multiple (3 or more) dysplastic nevi. - Current Bowen’s Disease, basal cell carcinoma, squamous cell carcinoma or other malignant skin lesions. - PLE currently requiring treatment with systemic immunosuppressive agents. - Documented history of other photosensitive conditions which may be confused with PLE or interfere with the assessment of PLE episodes. - Solarium use in the three months prior to study involvement and throughout the duration of the study. - Use of immunosuppressive medications, drugs that cause hyperpigmentation or any other treatment that in the opinion of the investigator may interfere with this study. - Documented presence (> 1 in 1000) of Anti Nuclear Antibody (ANA) and/or positive Extractable Nuclear Antibody (ENA); historical results from the 3 years prior to randomisation are acceptable if available. - Any evidence of clinically significant organ dysfunction, or any clinically significant deviation from normal in clinical or laboratory parameters. - History of drug or alcohol abuse (in the last 1 year). - Female who is pregnant (confirmed by positive serum β-HCG pregnancy test prior to baseline) or lactating. - Females of child-bearing potential (pre-menopausal, not surgically sterile) that are not using adequate contraceptive measures (e.g. oral contraceptives, condoms, diaphragm plus spermicide, intrauterine device). - Sexually active men with partners of child bearing potential not using barrier contraception during the trial and for a period of three months hereafter. - Participation in a clinical trial with another IMP within 30 days prior to the screening visit or during the study. - Hypersensitivity to afamelanotide or any of its components. - Hyperpigmentary disorders. - Use of therapeutic doses of β-carotene (in the last 3 months). - Use of any PLE medication/treatment (in the last 3 months).
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy Endpoints:
Primary Efficacy Endpoint: Physician’s Global Severity
Secondary Efficacy Endpoint: - Number of documented episodes of PLE - Quantity of rescue medication used - Quality of life using DLQI
Safety and Tolerability Endpoints: Primary Safety Endpoint: - Type and incidence of treatment-emergent adverse events.
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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 | Yes |
E.6.3 | Therapy | No |
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) | 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 | Yes |
E.8.1.6 | Cross over | Yes |
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.8.3 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 7 |
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 | 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
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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 | 2 |
E.8.9.1 | In the Member State concerned days | |
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 | 2 |