Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria
Conditions
Sponsors
Clinuvel Pharmaceuticals Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible to enter the study, patients must meet the following inclusion criteria: - Male or female subjects with a positive diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) - Aged 18-70 years (inclusive) - Provide written Informed Patient Consent prior to the performance of any study-specific procedure. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: To be eligible to enter the study, patients must not meet any of the following exclusion criteria: - Allergy to afamelanotide or the polymer contained in the implant or to lignocaine or other local anaesthetic to be used during the administration of the study medication - EPP patients with significant hepatic involvement - Personal history of melanoma or dysplastic nevus syndrome. - Current Bowen’s disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions. - Any other photodermatosis such as PLE, DLE or solar urticaria. - Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations. - Acute history of drug or alcohol abuse (in the last 12 months). - Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history, allergic to local anaesthetics, faints when given injections or giving blood). - Female who is pregnant (confirmed by positive serum ß-HCG pregnancy test prior to baseline) or lactating. - Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, 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 thereafter. - Participation in a clinical trial for an investigational agent within 30 days prior to the screening visit. - Prior and concomitant therapy with medications which may interfere with the objectives of the study, including drugs that cause photosensitivity or skin pigmentation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPP.;Secondary Objective: - To determine whether afamelanotide can reduce the number of phototoxic reactions in patients with EPP - To evaluate the safety and tolerability of afamelanotide by measuring treatment-emergent adverse events (AEs) - To determine whether afamelanotide can improve the quality of life of EPP patients - To determine the effect of afamelanotide on free protoporphyrin IX levels;Primary end point(s): -Efficacy Endpoints: Efficacy will be assessed by: - Number and severity of phototoxic reactions - Quality of life measured with DLQI questionnaire and supplementary EPP specific questions - Free protoporphyrin IX in peripheral erythrocytes Safety and Tolerability Endpoints: Treatment-emergent adverse events (coded as MedDRA Preferred Terms). Changes in hematology, serum chemistry and urinalysis measurements from Screening to Study Days 60, 120 and 180. | — |
Countries
Finland, Ireland, Netherlands, United Kingdom
Outcome results
None listed