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 | 12.1 |
E.1.2 | Level | LLT |
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 objectives - To determine if afamelanotide can reduce the severity of PLE related pruritis.
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E.2.2 | Secondary objectives of the trial |
Secondary objectives - To determine if afamelanotide can reduce the frequency of PLE episodes; - To determine if afamelanotide can reduce the duration of PLE episodes; - To evaluate the effect of afamelanotide on the use of rescue medication for the treatment of PLE episodes; - To evaluate the effect of afamelanotide on the quality of life of PLE patients; and - To evaluate the safety and tolerability of afamelanotide by measuring treatment-emergent adverse events.
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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, subjects must meet the following inclusion criteria: - Aged greater than 18 years; - Male or female patients with a documented history of PLE diagnosed or confirmed by a photodermatologist or photobiologist, with a history of PLE related pruritus symptoms; - Recurrent PLE episodes that occur at least once a year (as evidenced by PLE related pruritus symptoms) developing in their own country (to exclude patients affected only when traveling to sunnier climates); - Written informed consent prior to the performance of any study-specific procedure; and - 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, subjects 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; - Patients 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 320) 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; - In the opinion of the investigator, 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 or are not willing to use adequate contraceptive measures (e.g. oral contraceptives, condoms, diaphragm plus spermicide, intrauterine device); - Sexually active men with partners of child bearing potential not willing to use 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; and - Use of therapeutic doses of β-carotene and other excluded medicines, as described in the study protocol (in the last 3 months).
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Endpoints will be assessed by: - Severity of PLE related pruritus using an 11 point Likert scale, as recorded in the patient’s paper diary.
Secondary Endpoints will be assessed by: - Frequency (number) of documented episodes of PLE, as recorded in the patient’s paper diary; - Duration of PLE episodes; - Quantity of rescue medication used; - Quality of life using the Dermatology Life Quality Index (DLQI); and - The treatment-emergent adverse events (TEAEs), including clinically significant changes in laboratory parameters following 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 | 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 | 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.8.3 |
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 | 6 |
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 | 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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The end of the trial is defined by 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 | 0 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |